February 2026 Newsletter

Newsletters

A Message to Our Community

Dear Friends,

As we look ahead to spring, we are filled with renewed energy and optimism for the months to come. At NNPDF, our focus remains firmly on the future and on strengthening the support we provide to our entire Niemann-Pick disease community.

We are also investing in the future leadership of our organization. NNPDF is currently accepting applications for the Executive Director and Family Services Manager positions. These roles are vital to expanding our reach, strengthening family support services, and advancing our mission. We look forward to welcoming passionate leaders who will help guide NNPDF into its next chapter.

Thank you to everyone who joined our Community Connections Chats this month! Your participation, openness, and shared experiences are what make these conversations so meaningful. Topic suggestions are always welcome and encouraged for future chats! Your voice helps shape these discussions and ensures they remain valuable to our community.

As we continue building toward the future, our Niemann-Pick Toolkit remains a central priority. This important initiative is progressing steadily and will provide much-needed support through the development of a comprehensive Niemann-Pick Disease Toolkit. Designed to offer clearer direction, practical resources, and guidance, this toolkit will help families feel more informed and supported throughout their journey.

Respectfully,
The NNPDF Team

Rare Disease Day is February 28th!

Rare Disease Day is the globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.

Rare Disease Day promotes raising awareness and generating change for the 300 million people worldwide living with a rare disease such as Niemann-Pick, their families and caregivers. YOU can show your support on Rare Disease Day by raising awareness of Niemann-Pick Disease and what it means to be rare. Learn more and get involved!

Kasey Apt

Mom of Tinley, ASMD

Tell us a bit about you and your family…

Hi everyone! I am Kasey Apt and my daughter Tinley (ASMD) is 10 years old. Our family lives in a little town called Montpelier, Ohio… out in the middle of nowhere. We love going to the lake together as a family, going to hockey games, and serving in our church together. My husband, Thane, and I have three children: Tinley and her siblings, Addaley is 8 and Brentley who is almost 12. We also have a furry pup named Chett, who is Tinley’s very best friend.

Sharing Your Family’s Diagnosis Journey

When Tinley was about 3 years old, she had an enlarged belly, and my mama heart knew something wasn’t right. So, I asked her family doctor run a few simple tests to check for celiac disease or similar conditions, especially since her cousin had just been recently diagnosed with celiac. It didn’t come back positive for that, but the doctor could definitely tell something was still wrong… READ MORE

Continuing the Conversation

We are so grateful to everyone who participated in our Community Connections Chats this month! We especially want to thank our hosts, including community members, NNPDF Board members, and Scientific Advisory Board members, for sharing their time, insights, and expertise while leading such engaging conversations. These chats provide a positive and supportive space to connect, collaborate, share experiences, and ask questions. The camaraderie and thoughtful dialogue continue to strengthen and uplift our community.

We welcome and encourage your ideas and suggestions for future topics and special guests. Your participation and engagement help shape these conversations and ensure they remain meaningful and relevant for all. We look forward to gathering again soon and continuing these meaningful conversations together.

International Niemann-Pick Disease Registry

The INPDR is pleased to share the newly published Forum Report, which brings together key insights, priorities, and shared experiences from across the global Niemann‑Pick community. Over the past year, the Registry has strengthened its international research network through new clinical site partnerships and expanded global participation. The launch of the NP GUID further enhances secure, ethical collaboration by enabling consistent pseudonymised identifiers across studies, supporting researchers worldwide in advancing Niemann‑Pick… READ MORE

YARR Leadership Academy

The Young Adult Rare Representatives (YARR) Leadership Academy is now accepting applications for its Spring 2026 cohort, a six-week online program teaching young adults (ages 18–30) about federal advocacy, patient representation, and leadership in the rare disease space. Students will hear from expert speakers, including patient advocates, policymakers, and past Academy graduates, and learn how to have a meaningful impact on public policy. Classes run Thursdays from April 23 to June 4, and attendance at all six sessions is required to graduate. Applications are open through March 10. Learn more and apply.

Team Impact – Get Your Child In The Game
Team IMPACT is a national nonprofit that matches children facing serious illnesses and disabilities with college sports teams, creating a long-term, life-changing experience for everyone involved. By becoming an official member of a team, children gain a sense of belonging and empowerment, while student-athletes develop deep empathy and leadership skills through these unique mentorships. You can learn more about their incredible clinical model and how to get involved by visiting their website at teamimpact.org.

Special thanks to Dawn S. for sharing this helpful tip. If you have a resource or insight you’d like to share, we’d love to hear from you! Please send it to [email protected].

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please call 877-287-3672 or email [email protected] if you have any questions about this program.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by Niemann-Pick disease. Please continue to host and support Niemann-Pick fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

THANK YOU to Lynn Ell and Tauni Daub, who recently held a fundraiser to help support NNPDF’s mission!

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

ASMD and NPC Care Clinics

NNPDF has created a listing (formerly Comprehensive Care Centers) of U.S. academic and private practice clinics with experience in caring for and treating ASMD and NPC. We are pleased to offer this valuable resource to our patients and families as they search for diagnosis, medical care, and treatment.

Interested in joining our Care Clinics listing? The information in this listing is submitted directly by the medical providers. To have your clinic included, or if you are connected with a medical team that would like to be added, please visit our Care Clinics page for application information.

Plain Language Summaries

NNPDF is pleased to offer Plain Language Summaries to help our community stay informed about the latest medical research. These summaries translate complex scientific findings and clinical trial results into clear, accessible language, empowering patients and families to better understand advancements in Niemann-Pick care and treatment. This resource is designed to support more informed discussions with your healthcare team and help you make the best decisions for your family. Visit our Plain Language Summaries page to explore the latest updates.

Milestones

The NNPDF loves to showcase the special moments, memories, and meaningful steps you’ve been a part of along the way. If you have a special milestone to share with our Niemann-Pick community, send a photo along with the details to [email protected] and we’ll publish it in future communications!

Get Your NNPDF Gear and Awareness Apparel for Rare Disease Day

Now is a great time to get your Niemann-Pick Awareness apparel, NNPDF Logo apparel and NNPDF logo products shipped directly to your door! Order today and support your Niemann-Pick community! Store ships to U.S. residents only.

NIEMANN-PICK AWARENESS APPAREL is available! Raise awareness for ASMD or NPC, and even personalize for your family. Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options available. Send us your photos with your new gear!

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact us at [email protected]

With gratitude to our Funding Partners

We are grateful to these companies for their trust and support in advancing our mission.

Supporting one another. Supporting our community.

January 2026 Newsletter

Newsletters

A Message to Our Community

Dear Friends,

NNPDF remains dedicated to supporting patients and families affected by Niemann-Pick disease. Our focus is on providing the resources, connections, and trusted information you need. We are here to strengthen this community, uplift your voices, and ensure you feel supported throughout your journey.

Looking ahead, we are moving forward with a renewed sense of purpose. To that end, we’re growing our staff in leadership and family services to give this community dedicated care.

We have a busy year planned, starting in February with community chats and webinars leading up to Rare Disease Day on February 28, when we join the global community in recognition of the rare disease journey. Preparations are also underway for our annual Family Support & Medical Conference in Orlando, taking place July 9-11, bringing families, clinicians, and researchers together once again. In addition to the conference, we will continue to host Family Events throughout 2026, offering more opportunities for families to connect in smaller settings.

Together, we’ll continue showing up for one another, building connections, and moving forward as a strong, united community.

Respectfully,
The NNPDF Team

Jesika Coulbern

Mom of Kayden, NPC

Tell us a bit about you and your family…

Hello everyone! My name is Jesika Coulbern. My son, Kayden Smith, is 13 years old and his dad’s name is Justin Smith. Kayden is our only child and has our whole heart. We live in Indiana and have our whole life. We love going on walks and being outside as much as we can. Kayden love’s the ocean so we have made it a goal to go to as many aquariums as we can. We also were able to go to Hawaii for his make a wish trip and the smile that put on his face was everything.

Kayden loves walking, movies, car rides, swinging, bouncing balls, balloons, and popits. He’s overall just a happy kid that is so easy to fall in love with. I work at a hospital as a tech and love my job. I’ve always known I wanted to help children in healthcare, and I’ve been in the field for over… READ MORE

Rare Disease Day is February 28th!

Rare Disease Day is the globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.

Rare Disease Day promotes raising awareness and generating change for the 300 million people worldwide living with a rare disease such as Niemann-Pick, their families and caregivers. YOU can show your support on Rare Disease Day by raising awareness of Niemann-Pick Disease and what it means to be rare. Learn more and get involved!

NNPDF Member Survey

We want to extend a huge thank you to those in our community who have already shared their thoughts through our NNPDF Member Survey. We truly appreciate the time you took to provide such meaningful insights.

If you haven’t had a chance to participate yet, we’d still love to hear from you! Your experiences are the most valuable tools we have to ensure NNPDF continues to provide the specific support and resources for you and your family. Your anonymous responses help us demonstrate the real-life impact of our mission to the partners who make this work possible. We are truly grateful for your time, your patience, and your continued partnership.

Bringing Holiday Cheer Update

We are happy to share that 48 NNPDF community families received support to help make the holiday season a little brighter through Bringing Holiday Cheer! This program helps ease the burden of holiday stress, while promoting family togetherness and enjoyment, by helping to bring some cheer to our Niemann-Pick families. NNPDF was able to assist with over 500 items for our families.

We extend our deepest gratitude to our anonymous donor who started the gift giving program six years ago and for the generosity to all that contributed to this program. Thank you again for providing much joy to our families.

Wheelchairs 4 Kids is a nonprofit organization dedicated to improving the lives of children with physical disabilities and their families. Through its Let’s Roll Program, the organization provides essential equipment such as wheelchairs and home or vehicle modifications at no cost to families. These services help create safer, more supportive environments for children and their caregivers. Visit wheelchairs4kids.org for more information.

Special thanks to Dawn S. for sharing this helpful tip. If you have a resource or insight you’d like to share, we’d love to hear from you! Please send it to [email protected].

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please call 877-287-3672 or email [email protected] if you have any questions about this program.

Surveys, Studies & Market Research

NPC Patient Services Market Research Study

Mandos by Beren Therapeutics would like to make the NPC Community aware of the opportunity to share your experience and participate in this market research study.

The purpose of this market research is to better understand the experience and needs of NPC patients who are currently taking commercially approved therapies and their caregivers to help inform the development of a patient support service program for a potential new therapeutic option. Mandos Health by Beren Therapeutics is seeking participants for one-on-one interviews to share their perspectives. Learn more about this survey.

NPC Brain Imaging Research Study for NPC

Dr. Jaymin Upadhyay and his team at Boston Children’s Hospital are leading a research study to better understand NPC through advanced brain imaging and non-invasive assessments. The study includes questionnaires, cognitive and motor tasks, a blood sample, and non-invasive brain and muscle assessments. Visits take place over two days at Boston Children’s or McLean Hospital, with home visit options and travel support available. Participants will be compensated for their time. Learn more about this study.

Community News Updates

Update from IntraBio
IntraBio is pleased to announce that the European Commission has granted marketing authorization for AQNEURSA for the treatment of Niemann-Pick disease Type C (NPC). Following a positive opinion from the EMA’s CHMP, the therapy is approved in the European Union for use in adults and children aged 6 years and older weighing at least 20 kg. This milestone represents a significant step forward for the European NPC community. Read the complete announcement.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by Niemann-Pick disease. Please continue to host and support Niemann-Pick fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

THANK YOU to Kasey Apt who recently held a fundraiser to help support NNPDF’s mission!

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

ASMD and NPC Care Clinics

NNPDF has created a listing (formerly Comprehensive Care Centers) of U.S. academic and private practice clinics with experience in caring for and treating ASMD and NPC. We are pleased to offer this valuable resource to our patients and families as they search for diagnosis, medical care, and treatment.

Interested in joining our Care Clinics listing? The information in this listing is submitted directly by the medical providers. To have your clinic included, or if you are connected with a medical team that would like to be added, please visit our Care Clinics page for application information.

Plain Language Summaries

NNPDF is pleased to offer Plain Language Summaries to help our community stay informed about the latest medical research. These summaries translate complex scientific findings and clinical trial results into clear, accessible language, empowering patients and families to better understand advancements in Niemann-Pick care and treatment. This resource is designed to support more informed discussions with your healthcare team and help you make the best decisions for your family. Visit our Plain Language Summaries page to explore the latest updates.

Milestones

The NNPDF loves to showcase the special moments, memories, and meaningful steps you’ve been a part of along the way. If you have a special milestone to share with our Niemann-Pick community, send a photo along with the details to [email protected] and we’ll publish it in future communications!

Get Your NNPDF Gear and Awareness Apparel for Rare Disease Day

Now is a great time to get your Niemann-Pick Awareness apparel, NNPDF Logo apparel and NNPDF logo products shipped directly to your door! Order today and support your Niemann-Pick community! Store ships to U.S. residents only.

NIEMANN-PICK AWARENESS APPAREL is available! Raise awareness for ASMD or NPC, and even personalize for your family. Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options available. Send us your photos with your new gear!

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact us at [email protected]

With gratitude to our Funding Partners

We are grateful to these companies for their trust and support in advancing our mission.

Supporting one another. Supporting our community.

December 2025 Newsletter

Newsletters

A Message from our Board Chair

Dear Friends,

As the year draws to a close, on behalf of the Board of Directors and our organization, I extend our warmest holiday greetings to each of you. This season offers a meaningful opportunity for reflection, gratitude, and connection, and we are deeply thankful for the strong and supportive community that surrounds us.

Over the past few months, families have come together at our family gatherings to share laughter, stories, and meaningful moments. These occasions have been a beautiful reminder that we have built more than an organization, we have created a family. Bringing families together, strengthening relationships, and creating lasting memories remains at the heart of what we do. It has been truly heartwarming to witness the joy and sense of belonging experienced by so many.

We are also proud of the continued impact of our Holiday Cheer Program, which once again is bringing smiles, warmth, and joy to families during this special season. We extend our sincere appreciation to our dedicated staff for their hard work, commitment, and support during this especially busy time of year.

Looking ahead to the new year, we are enthusiastic about the opportunities before us. We remain committed to strengthening our programs and expanding our capacity to serve families in meaningful and impactful ways. We are confident that the year ahead will bring renewed energy, innovation, and continued growth.

On behalf of the Board of Directors, thank you for your continued trust, involvement, and support. We wish you and your loved ones a joyful holiday season filled with peace, warmth, and togetherness, and a happy, healthy New Year.

Warmly,

Liz Heinze, RN
NNPDF Board Chair

We Remember Them

As we reflect on 2025, we take a moment to remember the loved ones we’ve lost and to honor the courage of those who continue to carry their legacy. During this holiday season and into the new year, the NNPDF invites you to hold all Niemann-Pick families in your hearts, offering them your thoughts, support, and encouragement.

Community Update Webinar

NNPDF will host a community webinar featuring Mandos LLC, a subsidiary of Beren Therapeutics, on January 8, 2026, at 8:00 PM ET. The session will update the NPC community on the recent New Drug Application (NDA) submission to the FDA for adrabetadex (VTS-270) and provide an overview of the Early Access Program. Pre-registration is required.

Milestones

Congratulations to Emma! Emma (NPC) is a Junior at Culver High and has just received her letterman’s jacket, lettering in Music and Ballet. This is a great achievement that highlights her dedication, talent, and many hours of hard work. Her commitment to both music and ballet is truly impressive! Way to go Emma and well done!!

The NNPDF loves to showcase the special moments, memories, and meaningful steps you’ve been a part of along the way. If you have a special milestone to share with our Niemann-Pick community, send a photo along with the details to [email protected] and we’ll publish it in future communications!

Plain Language Summaries

NNPDF is pleased to offer Plain Language Summaries to help our community stay informed about the latest medical research. These summaries translate complex scientific findings and clinical trial results into clear, accessible language, empowering patients and families to better understand advancements in Niemann-Pick care and treatment. This resource is designed to support more informed discussions with your healthcare team and help you make the best decisions for your family. Visit our Plain Language Summaries page to explore the latest updates.

ASMD and NPC Care Clinics

NNPDF has created a listing (formerly Comprehensive Care Centers) of U.S. academic and private practice clinics with experience in caring for and treating ASMD and NPC. We are pleased to offer this valuable resource to our patients and families as they search for diagnosis, medical care, and treatment.

Interested in joining our Care Clinics listing? The information in this listing is submitted directly by the medical providers. To have your clinic included, or if you are connected with a medical team that would like to be added, please visit our Care Clinics page for application information.

Coming Soon! Tools for Meaningful Conversations

We’re excited to share that NNPDF is partnering with Be Clear, a communications firm that helps organizations tell their stories authentically to drive understanding and change. Together, we’re developing a new Family Storytelling Toolkit to support families navigating critical conversations with schools, insurers, clinicians, and more. This project reflects a core goal of our mission: giving families the tools, language, and confidence to advocate for the care and resources they deserve. The work is just beginning, and we can’t wait to share more with you soon.

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please call 877-287-3672 or email [email protected] if you have any questions about this program.

Community News Updates

Updates from Mandos

Beren Therapeutics, through its subsidiary Mandos LLC®, is pleased to share they’ve submitted a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for adrabetadex (VTS-270). This application seeks Priority Review for the treatment of patients with infantile-onset NPC. This milestone reflects the dedication and extraordinary commitment of families affected by Niemann-Pick disease type C. Read the complete press release.

Mandos Health is pleased to share they’ve received Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for adrabetadex, an investigational drug for infantile-onset Niemann-Pick disease type C. Mandos is excited to continue to progress this program. Read the complete announcement.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by Niemann-Pick disease. Please continue to host and support Niemann-Pick fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

Surveys, Studies & Market Research

NPC Brain Imaging Research Study for NPC

Dr. Jaymin Upadhyay and his team at Boston Children’s Hospital are leading a research study to better understand NPC through advanced brain imaging and non-invasive assessments. The study includes questionnaires, cognitive and motor tasks, a blood sample, and non-invasive brain and muscle assessments. Visits take place over two days at Boston Children’s or McLean Hospital, with home visit options and travel support available. Participants will be compensated for their time. Learn more about this study.

NNPDF Gear and Awareness Apparel

Now is a great time to get your Niemann-Pick Awareness apparel, NNPDF Logo apparel and NNPDF logo products shipped directly to your door! Order today and support your Niemann-Pick community! Store ships to U.S. residents only.

NIEMANN-PICK AWARENESS APPAREL is available! Raise awareness for ASMD or NPC, and even personalize for your family. Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options available. Send us your photos with your new gear!

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact us at [email protected]

With gratitude to our Funding Partners

We are grateful to these companies for their trust and support in advancing our mission.

Supporting one another. Supporting our community.

November 2025 Newsletter

Newsletters

A Message from our Board Chair

Dear Friends,

This Thanksgiving, the NNPDF Board and staff send our warmest wishes to all families for a holiday filled with love, laughter, and cherished moments together. We are so grateful for the strength and resilience of our community, whose support and connection uplift every family we serve.

We’re excited to welcome Dr. Shante Antrom as NNPDF’s Interim Executive Director! She will lead an organizational assessment to strengthen capacity and guide improvements, while also supporting daily operations. In addition, Dr. Antrom will contribute to the search for a permanent Executive Director, helping ensure a smooth transition. We’re thrilled to have her leadership during this important time.

Be sure to apply for the Bringing Holiday Cheer Program by November 30! Thanks to the vision of an anonymous donor who wanted to spread a little extra holiday cheer to Niemann-Pick families, this program brings together joy, warmth, and a sense of community during the busy season. We are truly grateful to everyone who helps make it happen.

A heartfelt thank-you to all the families who joined us at the Regional Family Event in Richmond, VA. It was a day full of connection, learning, and community support. PERSEVERE? You bet! After a fire at the hotel event venue canceled our original plans, quick thinking, fast planning, and extra hands allowed us to move the event outdoors to the Richmond Zoo. The day turned out beautifully, and we deeply appreciate everyone who attended, shared their stories, and strengthened the bonds within our Niemann-Pick community. Special thanks to all who helped make the transition happen!

Thank you for your continued trust, care, and partnership. Together, we move forward with hope, heart, and holiday cheer for every family we serve.

Warmly,

Liz Heinze, RN
NNPDF Board Chair

Bringing Holiday Cheer

November 30th is the final day to apply for the Bringing Holiday Cheer Program. We’d love you to take part in making this holiday season brighter. We remain dedicated to helping make the season a little less stressful and more meaningful for families in our community.

All NNPDF members residing in the U.S. are eligible to apply for the Bringing Holiday Cheer Program. Whether you are finding joy in the season, navigating the challenges of Niemann-Pick, or remembering a loved one, you are part of this community and are always remembered.

A heartfelt thank-you to everyone who helps make this program possible and look forward to sharing the holiday spirit with your families once again.

Application deadline is Sunday, November 30

Celebrating #GivingTuesday

On Tuesday, December 2nd, the National Niemann-Pick Disease Foundation will take part in #GivingTuesday, a day recognized across the nation for its spirit of giving. Your contribution can make a significant impact on families affected by Niemann-Pick Disease by enhancing the essential program services that NNPDF provides to our community. Please consider a gift on #GivingTuesday.

Supporting One Another. Supporting Our Community.

The Assistance Fund 2026 Reenrollment

2026 reenrollment is now open. If you are you currently receiving financial assistance through The Assistance Fund’s ASMD financial assistance program, please be aware of key dates and TAF’s reenrollment process so you are prepared to submit a reenrollment application for 2026 TAF assistance. Find complete details.

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please call 877-287-3672 or email [email protected] if you have any questions about this program.

Milestones

Tucker (NPC), an 11th-grader from Edgewood-Colesburg, had a lot to cheer for this season as his high school football team put together an incredible run. They dominated district play with a perfect 10–0 record and pushed through the state playoffs to finish an impressive 11–1. It was a proud, high-energy season that brought the whole community together and gave Tucker plenty of moments to celebrate. Way to go Tucker!!

The NNPDF loves to showcase the special moments, memories, and meaningful steps you’ve been a part of along the way. If you have a special milestone to share with our Niemann-Pick community, send a photo along with the details to [email protected] and we’ll publish it in future communications!

Fundraising Events

Pampered Chef Fundraiser for NNPDF

Kasey Apt hosted an online fundraiser to help raise awareness during Niemann-Pick Awareness Month, and it turned out to be a wonderful success. The Pampered Chef fundraising event drew tremendous support. Kasey shared, “The foundation is so special to us! They have been there for us from the very beginning of finding out about Tinley’s diagnosis. We wanted to show our appreciation by giving back a little!”

Thank you to Kasey and to everyone who ordered those must-have Pampered Chef kitchen items.

Smoothie King Fundraiser for NNPDF

Every Tuesday during Niemann-Pick Awareness Month the Florkiewicz family collected a percentage of their smoothie sales to donate back to the NNPDF in Kailey’s honor.

Krissy shared “Thanks to all that participated and supported us in making this event a huge success. Kailey was diagnosed with Niemann-Pick type C on February 16, 2013, when she was 9 years old. Since that time, she has participated in every drug trial available to her to help find a cure. There is still no cure. Kailey has lost her ability to speak, eat by mouth, and uses a wheelchair all of the time now. She still tries hard and fights hard every day.

We fundraise when we can to help research for a cure. I still hope to see a cure in Kailey’s lifetime. Thank you all for donating and keeping K in your thoughts and prayers every day.”

Thank you Krissy and to all those who stopped in and supported the Florkiewicz family.

19th Annual NNPDF Dillon Papier Baysox Benefit

DARRILE PAPIER – The 19th Annual NNPDF Dillon Papier Baysox Benefit was held on Sunday, August 24th, bringing together more than 230 friends and family for an afternoon of celebration, community, and fun. Guests enjoyed delicious food and participated in over 99 raffle drawings throughout the event. It was an exciting day, highlighted by Caleb Woodard and Liam Ferguson throwing out the ceremonial first pitch on behalf of NNPDF, a heartfelt tribute to Dillon’s love of baseball.

Also in attendance were Dr. Porter, Sherri, and the Ferguson and Woodard families, who rarely miss a benefit. Nicole Farhat, NIH and her family also joined in, enjoying the festivities and a fun NNPDF mini reunion filled with laughter, connections, and shared memories. The event was a wonderful celebration and reminder of the strength and spirit that define the NNPDF community. We’re so grateful to everyone who helped make the day such a success!

NIEMANN-PICK AWARENESS – We were also thrilled to visit the NIH Children’s Inn on Dillon’s birthday and on the anniversary of his passing, as the amazing staff there have become part of our extended family. We were thrilled to be granted special permission to bake onsite and we might have gone a little overboard as we baked 8 different cookie flavors, 3 varieties of mini bundt cakes and brownies, totaling 314 baked goodies! It was such a joy to see the smiles on the kids and families’ faces as they sampled the treats some even got to taste test while the cookies were still cooling before we package them up to share.

THANK YOU to Barbara Lazarus and Anne OConnor Smith who recently held Facebook Fundraisers to help support NNPDF’s mission!

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by Niemann-Pick disease. Please continue to host and support Niemann-Pick fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

Surveys, Studies & Market Research

NPC Brain Imaging Research Study for NPC

Dr. Jaymin Upadhyay and his team at Boston Children’s Hospital are leading a research study to better understand NPC through advanced brain imaging and non-invasive assessments. The study includes questionnaires, cognitive and motor tasks, a blood sample, and non-invasive brain and muscle assessments. Visits take place over two days at Boston Children’s or McLean Hospital, with home visit options and travel support available. Participants will be compensated for their time. Learn more about this study.

Clinical Trial Updates

Clinical trials are currently in progress to study and develop treatments for ASMD and NPC. The NNPDF posts new information regarding clinical trial updates as soon as it is received. Visit our Clinical Trials web page for up to date information on all clinical trials.

Update from Azafaros:
Azafaros is pleased to share the opening of the first U.S. study site for the Phase 3 NAVIGATE clinical trial, sponsored by Azafaros. This double-blind, placebo-controlled study evaluates the safety and efficacy of nizubaglustat for treating late-infantile/juvenile NPC and GM1/GM2 diseases. Learn more.

NNPDF Gear and Awareness Apparel

Now is a great time to get your Niemann-Pick Awareness apparel, NNPDF Logo apparel and NNPDF logo products shipped directly to your door! Order today and support your Niemann-Pick community! Store ships to U.S. residents only.

NIEMANN-PICK AWARENESS APPAREL is available! Raise awareness for ASMD or NPC, and even personalize for your family. Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options available. Send us your photos with your new gear!

Comprehensive Care Centers

NNPDF provides a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. Click here to learn more!

If you are connected with a medical team that would like to be added to the NNPDF Comprehensive Care Centers listing, please email [email protected].

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact us at [email protected]

OUR VALUED PARTNERS

We are grateful to these companies for their trust and support in advancing our mission.

Supporting one another. Supporting our community.

October 2025 Newsletter

Newsletters

A Message from our Board Chair

Dear Friends,

The fall season brings change, but one thing that remains constant is the strength and warmth of our NNPDF community. As we wrap up October Awareness Month, I want to thank you for helping us raise awareness by sharing our communications, joining our Community Chats, and supporting one another. Your engagement truly makes a difference. We also welcome your ideas for future chat topics that matter most to you and your families.

The Board of Directors continues to guide the organization through this time of transition, focused on keeping our programs steady and our families supported. We will be welcoming an interim Operations Manager to provide leadership while we thoughtfully search for our next Executive Director. Hiring a Family Services Manager also remains a top priority as we respond to the evolving needs of our community.

We hope your family will join us for the Regional Family Event on November 8 in Richmond, VA. Registration closes October 30. It will be a wonderful day to gather, learn, connect, and share with others in your local Niemann-Pick community. Come meet other families, hear updates, and enjoy a day of support and connection.

Looking ahead, we are also excited to share our annual Bringing Holiday Cheer Program, started by an anonymous donor who wanted to make the holidays a little brighter for Niemann-Pick families. This program helps bring a sense of togetherness and joy during the busy season, and we are so grateful to everyone who continues to make it possible.

We’re happy to share that the photos from our 2025 conference are now complete, and all event photos and recordings are available on our Family Support & Medical Conference page. Thank you again to the over 200 family members, friends, and colleagues who attended and contributed to such a meaningful event. Remember to save the date for next year’s conference from July 9 to 11 in Orlando, FL.

Thank you for your continued trust, care, and partnership. Together, we will keep moving forward with hope and heart for every family we serve.

Warmly,

Liz Heinze, RN
NNPDF Board Chair

NNPDF Family Event: You're Invited to Richmond, VA!

November 8 | Richmond, VA

Registration Deadline is October 30th

We’re excited to welcome you to the NNPDF Family Event on Saturday, November 8. This special day brings together Niemann-Pick families from the greater Richmond area to connect and support one another.

Spend the day sharing experiences, learning, and enjoying meaningful time with others in your community. We look forward to seeing you and celebrating together!
This is a free event for NNPDF immediate family members and lunch will be provided.
Pre-registration is required for all attendees by October 30th.

Bringing Holiday Cheer

NNPDF is excited to share that the Bringing Holiday Cheer program is now underway! In its 6th year, this program was founded by an anonymous donor and continues to brighten the holidays for our Niemann-Pick families, helping make the season a little brighter and creating special memories together. Thank you to everyone who helps make this tradition possible!

All NNPDF members residing in the U.S. are eligible to apply for the Bringing Holiday Cheer Program. Whether you are finding joy in the season, navigating the challenges of Niemann-Pick, or remembering a loved one, you are part of this community and are always remembered.

Application deadline is Friday, November 15th

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please call 877-287-3672 or email [email protected] if you have any questions about this program.

Surveys, Studies & Market Research

NPC Brain Imaging Research Study for NPC

Dr. Jaymin Upadhyay and his team at Boston Children’s Hospital are leading a research study to better understand NPC through advanced brain imaging and non-invasive assessments. The study includes questionnaires, cognitive and motor tasks, a blood sample, and non-invasive brain and muscle assessments. Visits take place over two days at Boston Children’s or McLean Hospital, with home visit options and travel support available. Participants will be compensated for their time. Learn more about this study.

Milestones

The NNPDF loves to showcase the special moments, memories, and meaningful steps you’ve been a part of along the way. If you have a special milestone to share with our Niemann-Pick community, send a photo along with the details to [email protected] and we’ll publish it in future communications!

NNPDF Gear and Awareness Apparel

Now is a great time to get your Niemann-Pick Awareness apparel, NNPDF Logo apparel and NNPDF logo products shipped directly to your door! Order today and support your Niemann-Pick community! Store ships to U.S. residents only.

NIEMANN-PICK AWARENESS APPAREL is available! Raise awareness for ASMD or NPC, and even personalize for your family. Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options available. Send us your photos with your new gear!

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by Niemann-Pick disease. Please continue to host and support Niemann-Pick fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

THANK YOU to Eric Shawn Peterson and Justin Smitty who recently held a fundraiser to help support NNPDF’s mission!

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

Comprehensive Care Centers

NNPDF provides a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. Click here to learn more!

If you are connected with a medical team that would like to be added to the NNPDF Comprehensive Care Centers listing, please email [email protected].

ASMD Accelerate

The Assistance Fund

Struggling with out-of-pocket expenses due to acid sphingomyelinase deficiency (ASMD)? The Assistance Fund is an independent charitable patient assistance organization helping patients and families by providing financial assistance for the cost of treatment and other health-related services. Through its Acid Sphingomyelinase Deficiency Financial Assistance Program, eligible individuals can receive assistance for all FDA-approved treatment for ASMD. Learn more.

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact Laurie at [email protected] or call 920-542-4038

Supporting one another. Supporting our community.

September 2025 Newsletter

Newsletters

Your Voice Matters During Niemann-Pick Awareness Month

There is strength in community, and there is power in our voices. When we come together – families, friends, advocates, and allies – we shine a light on Niemann-Pick Disease. Awareness isn’t just about information, it’s about action, empathy, and standing in solidarity with every person and family impacted by this disease.

Each story shared, each conversation started, each life touched are the moments that build momentum. Our unity speaks louder than words and it’s something we build together, one voice at a time.

Show your support for the Niemann-Pick community by helping to raise awareness and educate others. Whether you’re sharing a post, telling your story, or simply listening and learning, you are part of a movement and reason someone learns about Niemann-Pick Disease for the first time, and the reason they choose to care.

During Niemann-Pick Awareness Month and beyond, let’s continue to raise our voices, uplift our community, and remind every family facing Niemann-Pick that they are not alone.

Supporting One Another. Supporting Our Community.

NNPDF Family Event: You're Invited to Richmond, VA!

November 8 | Richmond, VA

We’re excited to welcome you to the NNPDF Family Event on Saturday, November 8. This special day brings together Niemann-Pick families from the greater Richmond area to connect and support one another.

Spend the day sharing experiences, learning, and enjoying meaningful time with others in your community. We look forward to seeing you and celebrating together!
This is a free event for NNPDF immediate family members and lunch will be provided.
Pre-registration is required for all attendees by October 30th.

Community Connections During Awareness Month!

We’re excited to offer several virtual opportunities for NNPDF families to come together, connect, and support one another throughout the month. Whether you’re looking to catch up, learn, share, or simply take a meaningful break, we invite you to join us in strengthening our community bonds. Stay tuned for event dates and details coming soon!

Milestones

The NNPDF loves to showcase the special moments, memories, and meaningful steps you’ve been a part of along the way. If you have a special milestone to share with our Niemann-Pick community, send a photo along with the details to [email protected] and we’ll publish it in future communications!

Surveys, Studies & Market Research

NPC Brain Imaging Research Study for NPC

Dr. Jaymin Upadhyay and his team at Boston Children’s Hospital are leading a research study to better understand NPC through advanced brain imaging and non-invasive assessments. The study includes questionnaires, cognitive and motor tasks, a blood sample, and non-invasive brain and muscle assessments. Visits take place over two days at Boston Children’s or McLean Hospital, with home visit options and travel support available. Participants will be compensated for their time. Learn more about this study.

NNPDF Gear and Awareness Apparel

Now is a great time to get your Niemann-Pick Awareness apparel, NNPDF Logo apparel and NNPDF logo products shipped directly to your door! Order today and support your Niemann-Pick community! Store ships to U.S. residents only.

NIEMANN-PICK AWARENESS APPAREL is available! Raise awareness for ASMD or NPC, and even personalize for your family. Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options available. Send us your photos with your new gear!

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by Niemann-Pick disease. Please continue to host and support Niemann-Pick fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

THANK YOU to Sara Christophel who recently held a fundraiser to help support NNPDF’s mission!

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

Be Part of the Story: Submit Your Photos!

We’d love to feature the incredible strength and spirit of our community by including your photos in our communications and promotions throughout the year! Whether it’s a family photo or a picture of your Niemann-Pick warrior, your photo helps tell our story. By sharing them, you’ll help us spread awareness, build support, and highlight the power of our amazing community. Please send your favorite photos today to [email protected]!

Thank you for supporting our NNPDF community. We truly appreciate and THANK each and every one of you for all you do.

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please call 877-287-3672 or email [email protected] if you have any questions about this program.

Comprehensive Care Centers

NNPDF provides a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. Click here to learn more!

If you are connected with a medical team that would like to be added to the NNPDF Comprehensive Care Centers listing, please email [email protected].

ASMD Accelerate

The Assistance Fund

Struggling with out-of-pocket expenses due to acid sphingomyelinase deficiency (ASMD)? The Assistance Fund is an independent charitable patient assistance organization helping patients and families by providing financial assistance for the cost of treatment and other health-related services. Through its Acid Sphingomyelinase Deficiency Financial Assistance Program, eligible individuals can receive assistance for all FDA-approved treatment for ASMD. Learn more.

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact Laurie at [email protected] or call 920-542-4038

Supporting one another. Supporting our community.

Summer 2025 Newsletter

Newsletters

A Message from our Board Chair

Dear Friends,

Just over a month has passed since we gathered in person at our annual Family Conference and once again, I find myself missing our incredible NNPDF community. This year has brought its share of transitions and challenges, but your feedback after the conference has been deeply encouraging. It reminds us why we do what we do: to build connections, strengthen support, and foster relationships that help so many of us navigate everyday life with Niemann-Pick Disease.

As many of you know, our Executive Director stepped down just a few short weeks before the conference. While we are grateful for the contributions she made during her time with us, we understand and respect that this was not an easy decision. In the wake of her departure, our dedicated Board of Directors have been working diligently to move forward with intention and care. We are taking this time to thoughtfully evaluate the evolving needs of our community and to ensure that our next Executive Director brings not only the right experience, but also a heart for our mission and families.

Earlier this year, we also experienced another major transition as our beloved Laurie Turner announced her departure from NNPDF to pursue new aspirations as well as spend time with her family as they grow and achieve all that they are meant to be. Laurie’s impact on our community is immeasurable, and while we miss her presence dearly, we are grateful for the support she continues to offer during this transition. Her legacy of compassion and advocacy remains deeply embedded in the work we do every day.

Despite these changes, our commitment to serving you remains unwavering. The Foundation is strong and steady, thanks to the tireless efforts of our Board and staff. We continue to meet families wherever they are in their journey with Niemann-Pick Disease and strive to be a source of guidance, support, and hope.

The Family Support & Medical Conference recordings are now available! Whether you were able to join us in person or not, we hope these sessions offer valuable insights and strengthen the sense of connection that makes this community so special. Looking ahead, we are excited to launch new tools and resources to help you better understand and navigate this disease.

With October Awareness Month on the horizon, we have a full calendar of engaging content planned – from social media profile frames and community chats to impactful awareness campaigns. There will be many ways to get involved and help amplify our shared mission.

Thank you for your continued trust, resilience, and the love you bring to this community. Together, we are moving forward with purpose, with strength, and always with heart.

Warmly,

Liz Heinze, RN
NNPDF Board Chair

NNPDF Family Support & Medical Conference Recap

Thank you for being part of the 2025 NNPDF Family Support & Medical Conference! It was an inspiring weekend filled with learning, connection, and support among Niemann-Pick families, researchers, and healthcare professionals.

We’re incredibly grateful and extend a sincere THANK YOU to our volunteers, sponsors, and community partners for your dedication in making this wonderful event possible for our Niemann-Pick families and community members! It was great to see such strong support and encouragement of one another being shared.

The conference recordings are now available! If you have any questions or comments you’d like to share with us, please contact us at [email protected]. We look forward to seeing you again in Orlando, Florida in 2026!

Community Update Webinar

Join us for Community Update Webinar featuring:
Mandos Health® by Beren Therapeutics
September 8, 2024 at 8:00 pm ET

This event is exclusively for NNPDF community families living in the U.S. and Canada.

Preregistration is required and will be subject to approval.

Upcoming Family Events

We hope your family will join us for for a day to gather, learn, connect, and share with others in the Niemann-Pick community! Preregistration is required for all attendees and lunch will be provided.

Family Events are a special time for everyone to come together, so NNPDF will not have an Activity Zone at these events.

Registration and further details will be available soon – please keep an eye on your inbox!

CHICAGO, ILLINOIS
September 27, 2025 | 10:00 am – 4:00 pm CT
Lurie Children’s Hospital, Chicago, IL
RICHMOND, VIRGINIA
November 8, 2025 | 10:00 am – 4:00 pm ET
Location to be announced.

Surveys, Studies & Market Research

Surveys, studies, and market research play a critical role in deepening our understanding of the needs and experiences of individuals and families affected by Niemann-Pick disease. Your participation contributes essential data that informs scientific research, guides the development of treatments, enhances support services, and strengthens advocacy efforts across the community.

Learn more about the following surveys and ongoing studies actively accepting participants.

Understanding the management and unmet needs among individuals diagnosed with Niemann-Pick disease Type C in the US
This is a blinded market research study to understand current NPC patient management, treatment expectations, outcome measurement, and decisions around stopping or adding treatments. This is a 60 minute telephone interview where you will need access to a computer. Participants will be compensated for their time. Learn more about this study.

NPC Brain Imaging Research Study for NPC
Dr. Jaymin Upadhyay and his team at Boston Children’s Hospital are leading a research study to better understand NPC through advanced brain imaging and non-invasive assessments. The study includes questionnaires, cognitive and motor tasks, a blood sample, and non-invasive brain and muscle assessments. Visits take place over two days at Boston Children’s or McLean Hospital, with home visit options and travel support available. Participants will be compensated for their time. Learn more about this study.

Milestones

The NNPDF loves to showcase the special moments, memories, and meaningful steps you’ve been a part of along the way. If you have a special milestone to share with our Niemann-Pick community, send a photo along with the details to [email protected], and we’ll publish it in future communications!

Kelly

Kelly and Aaron celebrated Kelly’s 46th birthday in Bora Bora! Happy (belated) birthday Kelly!!

Emma

Emma attended a Music Festival in the Pacific Northwest with 300 other students. She had a blast! What a fun experience, Emma!

Cole

Cole graduated high school with honors on May 23rd! Congratulations Cole!

Victoria

Victoria celebrated her completion of 7th grade! It was a year of firsts and adjustments but the school team was amazing! Way to go Victoria!

Connor

Connor lettered in orchestra (violin) and in each of his three adaptive sports seasons. Congrats Connor! Go Mustangs!

Marian

Marian gave a graceful performance at the ballet recital. Beautifully done Marian!

Liam

Liam achieved the rank of Weblos in the Cub Scouts and earned his half red/ half black belt in Martial Arts. In addition, Liam’s matched baseball team (Marymount Saints) won their second conference championship! Well done Liam!

Arnold Family

The Arnold Family had several milestones this past year: Stella performed in her first dance recital! Roman made some great improvements in communication and physical strength! Jillian had her first children’s book published! Well done Arnold family!

Clinical Trial Updates

Clinical trials are currently in progress to study and develop treatments for ASMD and NPC. The NNPDF posts new information regarding clinical trial updates as soon as it is received. Visit our Clinical Trials web page for up to date information on all clinical trials.

Update from Azafaros:
Azafaros is pleased to share the dosing of the first patient in its pivotal Phase 3 NAVIGATE program. The two global studies will evaluate the safety and efficacy of the company’s lead asset, nizubaglustat, in individuals living with Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses. This marks a significant milestone in Azafaros’ mission to deliver new treatment options for patients affected by these neurodegenerative disorders. Read the complete announcement.

Community News Updates

Update from IntraBio
IntraBio is pleased to share that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending approval of AQNEURSA® (levacetylleucine) for the treatment of Niemann-Pick disease type C (NPC). Read the complete announcement.

Update from Zevra Therapeutics
Zevra Therapeutics is pleased to announce the filing of their Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for arimoclomol. This marks a major milestone in the company’s journey toward making MIPLYFFA foundational therapy available to patients with NPC across Europe. Read the complete announcement.

Be Part of the Story: Submit Your Photos!

We’d love to feature the incredible strength and spirit of our community by including your photos in our communications and promotions throughout the year! Whether it’s a family photo or a picture of your Niemann-Pick warrior, your photo helps tell our story. By sharing them, you’ll help us spread awareness, build support, and highlight the power of our amazing community. Please send your favorite photos today to [email protected]!

Thank you for supporting our NNPDF community. We truly appreciate and THANK each and every one of you for all you do.

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

If you have any questions about this program please reach out to us at [email protected] or 877-287-3672.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to host and support NPD fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

THANK YOU to Ashley Abare, Shawnae Sillanpaa, April Van Dyke, and Suzanne O’Leary who recently held a fundraiser to help support NNPDF’s mission!

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

Comprehensive Care Centers

NNPDF provides a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. Click here to learn more!

If you are connected with a medical team that would like to be added to the NNPDF Comprehensive Care Centers listing, please contact [email protected].

The Assistance Fund

Struggling with out-of-pocket expenses due to acid sphingomyelinase deficiency (ASMD)? The Assistance Fund is an independent charitable patient assistance organization helping patients and families by providing financial assistance for the cost of treatment and other health-related services. Through its Acid Sphingomyelinase Deficiency Financial Assistance Program, eligible individuals can receive assistance for all FDA-approved treatment for ASMD. Learn more.

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact [email protected] or call 877-287-3672

Supporting one another. Supporting our community.

May 2025 Newsletter

Newsletters

July 10-13, 2025

Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa
Concord, North Carolina

We look forward to seeing you at our 33rd Annual NNPDF Family Support & Medical Conference! Be sure to watch NNPDF communications for important updates. We look forward to seeing you there!

For all conference information visit nnpdf.org/conf.

Conference Registration

Conference Shirt Deadline – Register by June 10th to ensure your complimentary 2025 NNPDF Family Support & Medical Conference shirt will be waiting for you at the conference!

First Time Attendee Discount: Enjoy a 10% discount off your registration! We’re happy to have you join us!

June 28th is the final day for registration so please be sure to complete your registration by this important deadline.

Accommodations

Reserve by June 11th

Our 2025 conference will be held at the beautiful Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa in Concord, North Carolina. The NNPDF group rate will be available until June 11th, 2025. NNPDF Group Rate is $179 ($203.61 including taxes) per night. The NNPDF hotel group rate is available three days before and three days after the conference, dependent on hotel availability.

Special Offer for Conference Attendees

We are excited to share a special offer for Family Support and Medical Conference attendees.

Order your NNPDF gear by June 10th and take advantage of FREE SHIPPING when you pick up your merchandise at the NNPDF Family Support & Medical Conference!

It’s easy!

Select your gear and place your order
In Shipping Method select “PICK UP AT CONFERENCE for FREE”
Grab your gear at the conference!

I Need A Cure for NPD - ASMD - Store Image

Someone I Love Needs a Cure - NPC - Store Image

NNPDF Supports Newborn Screening Committee

NNPDF joins the broader rare disease community in signing on to preserve the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) federal-level committee focused on newborn screening. This committee plays a vital role in the early diagnosis of rare and serious genetic conditions in newborns, and its continued work is essential to the future of this important effort. We’re proud to stand with our partners in support of this initiative.

Read the community letter of support.

NPC Study Opportunity: Help us better understand the impact of living with NPC

Niemann-Pick type C (NPC) impacts the quality of life for both those living with the condition and their families and caregivers. If you have NPC or care for someone with NPC, we invite you to join our research study and share your experiences. Your participation is crucial in helping us better understand NPC and improve support for those affected.

Learn more and find out how to get involved. Your voice matters!

Courageous Parents Network Virtual Workshop

Courageous Parents Network invites you to join their upcoming virtual workshop: Parent Perspectives: Insights into Medical Decision-Making on Wednesday, June 25, 2025 at 12:00 PM ET.

Making decisions about interventions like feeding tubes or tracheostomies is one of the toughest responsibilities caregivers face. In this session, a panel of experienced parents will share what guided their choices, the challenges they faced, and how they worked with their care teams. Gain real-life insight and practical advice to support your own decision-making journey. Moderated by Gabrielle Conecker, a public health advocate with over 20 years of experience.
Register here!

Milestones

Do you have a special milestone to share with our Niemann-Pick Community? Please consider submitting milestone moments and family photos so we can showcase the special moments you have been a part of.

Send a photo along with the details to [email protected] and we’ll publish it in future communications!!

Community News Update

NNPDF shares news and information regarding our community as soon as it is received. Visit our Community News web page for up to date information.

Update from Azafaros: Azafaros announces its completion of an oversubscribed €132M Series B financing enabling accelerated development of its lead product nizubaglustat, scheduled to enter Phase 3 studies for Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses later this year. Read the complete update.

Be Part of the Story: Submit Your Photos!

We’d love to feature the incredible strength and spirit of our community by including your photos in our communications and promotions throughout the year! Whether it’s a family photo or a picture of your Niemann-Pick warrior, your photo helps tell our story. By sharing them, you’ll help us spread awareness, build support, and highlight the power of our amazing community. Please send your favorite photos today to [email protected]!

Thank you for supporting our NNPDF community. We truly appreciate and THANK each and every one of you for all you do.

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please contact [email protected] if you have any questions about this program.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to host and support NPD fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

THANK YOU to Suzanne O’Leary who recently held a Facebook Fundraiser!

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

ASMD Accelerate

The Assistance Fund

Struggling with out-of-pocket expenses due to acid sphingomyelinase deficiency (ASMD)? The Assistance Fund is an independent charitable patient assistance organization helping patients and families by providing financial assistance for the cost of treatment and other health-related services. Through its Acid Sphingomyelinase Deficiency Financial Assistance Program, eligible individuals can receive assistance for all FDA-approved treatment for ASMD. Learn more.

Comprehensive Care Centers

NNPDF provides a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. Click here to learn more!

If you are connected with a medical team that would like to be added to the NNPDF Comprehensive Care Centers listing, please contact [email protected].

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact [email protected] or call 877-287-3672.

Supporting one another. Supporting our community.

April 2025 Newsletter

Newsletters

Message from our Executive Director

Hi everyone,

A month into the role as your Executive Director, I’ve now met with every board member and scientific advisor, as well as our vendors, industry partners, and more. I’ve also begun hosting a series of Community Conversations (with families) and Clinical Conversations (with our doctors and their office staff) in an effort to build out some resources for our families that can support you through insurance and access barriers. As I learn more about what these hurdles and burdens look like, I’m interviewing experts and working on a three prong approach that should encompass casework support, toolkits and materials to get ahead of the problems, and policy advocacy to educate policymakers about improving the laws and regulations that allow these hurdles to exist in the first place.

If you haven’t yet signed up for one of the Community Conversations, please do. Watch for new sessions coming soon! Whether your insurance problems are behind you or you’re dealing with them now, it would help me to learn what worked as I create the resources and tools that might help.

I also can’t wait to meet you at the Family Support & Medical Conference in Concord, NC in July. As the staff pulls together another wonderful conference this year, I want to remind you to register! We have lots of great speakers, including a session on public policy and what is happening in Washington, D.C. Also, last call on signing up for the Family Advisory Working Groups and Conference Scholarships – final deadline is April 20th! Join us and make this year’s family conference a success!

Warm regards,

Saira Sultan, JD
NNPDF Executive Director

July 10-13, 2025

Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa
Concord, North Carolina

We look forward to seeing you at our 33rd Annual NNPDF Family Support & Medical Conference! Be sure to watch NNPDF communications for important updates. We look forward to seeing you there!

For all conference information visit nnpdf.org/conf.

Family Advisory Working Groups

Final Deadline April 20th

The National Niemann-Pick Disease Foundation, Inc. (NNPDF) will host Family Advisory Working Groups that will take place on on Thursday, July 10, 2025 prior to the start of our Family Support & Medical Conference at the Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa in Concord, North Carolina.

Family Advisory Working Groups facilitate communication between Niemann-Pick families and the pharmaceutical industry, offering valuable insights into the patient and family experience. These sessions provide industry partners with the opportunity to engage privately with a small group of ASMD or NPC patients and/or immediate family members for a 3-hour discussion. This is an optional event and pre-registration is required.

Family Conference Scholarships

Final Deadline April 20th

Scholarship funding is available to help NNPDF families ease the cost of attending the 2025 NNPDF Family Support and Medical Conference in Concord, North Carolina. Scholarship assistance will provide up to 3 nights hotel (room and tax only at the group rate) and up to 2 adult conference registrations for eligible applicants. Scholarship assistance is limited to immediate NNPDF U.S. family members of an individual with Niemann-Pick Disease.

Conference Registration

Early Bird Registration: Save by May 14th

Discounted Registration Rate:
Register by May 14th to take advantage of discounted registration fees.

Family Registration Discounted Rate:
EARLY BIRD REGISTRATION ONLY – Register 2 adults and 2 children (age 17 and younger) at the early bird registration rate of $500 (adults $175 and children $75). Any additional children in your immediate family will be free of charge.

First Time Attendee Discount:
Enjoy a 10% discount off your registration! We’re happy to have you join us!

Accommodations

Reserve by June 11th

Our 2025 conference will be held at the beautiful Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa in Concord, North Carolina. The NNPDF group rate will be available until June 11th, 2025. NNPDF Group Rate is $179 ($203.61 including taxes) per night. The NNPDF hotel group rate is available three days before and three days after the conference, dependent on hotel availability.

NNPDF Cora Sterling Endurance Award

Application Deadline May 15th

Do you know an inspiring young person who’s making a difference in raising Niemann-Pick awareness? Nominate them for the NNPDF Cora Sterling Endurance Award! This special award is presented annually at the NNPDF Family Support & Medical Conference to a remarkable individual under age 25 who shines in raising awareness of Niemann-Pick disease in the community. Don’t miss the chance to celebrate their efforts!

Joele Ruppert & Joseph Colton ASMD Scholarship

Application Deadline May 15th

NNPDF is accepting applications for the Joele Ruppert and Joseph Colton ASMD Scholarship. This scholarship provides one-time funding of $500 to individuals diagnosed with ASMD for studies in post-secondary (high school) degrees, including two-year colleges, four-year colleges, vocational schools, and other post-secondary institutions.

National Volunteer Month

Volunteers are essential in the progress of Niemann-Pick Disease awareness and your NNPDF organization!

During NATIONAL VOLUNTEER MONTH we acknowledge each of you who have generously given your time and energy to keep your NNPDF family support organization moving forward. We truly appreciate and THANK each and every one of you for all you do.

NNPDF Board of Directors volunteer selflessly to serve in these vitally important roles.
Scientific Advisory Board members advocate for your cause.
Family conference leaders and helpers give their time tirelessly and unconditionally.
Friends and family members volunteer to raise funds that contribute to Niemann-Pick disease research and support your family organization programs.
Families give their time in simply supporting one another through the trials and struggles that Niemann-Pick disease brings.

Your community is growing stronger because of you! Thank you!

Supporting One Another. Supporting Our Community.

Celebrating Leadership:

Justin Hopkin Appointed INPDR Chief Scientific Officer

We’re proud to congratulate Justin Hopkin on his new role as INPDR’s first-ever Chief Scientific Officer! Justin has long been a dedicated advocate for our Niemann-Pick families, and this exciting new chapter is a well-deserved recognition of his tireless commitment to advancing care, research, and community support.

As an INPDR Ambassador, member of the Niemann-Pick community, and now CSO, Justin brings both deep personal insight and scientific leadership to the role. Join us in celebrating this exciting achievement and all that it means for the future of the Niemann-Pick community.

#RAREis Scholarship Application Open

Living with a #RareDisease? Have a dream to pursue your education? Perhaps a $5,000 scholarship would help. The #RAREis Scholarship Fund is now accepting applications for 2025!

The #RAREis Scholarship Fund is available for any rare disease patient who is age 17+ and has a U.S. residency. Applicants must be enrolled in an accredited course(s) for Fall 2025 Semester. Therefore, it is not degree specific!

If financial burden has held you back before, now is the time to pursue those dreams! Learn more.

NPC Study Opportunity: Help us better understand the impact of living with NPC

Niemann-Pick type C (NPC) impacts the quality of life for both those living with the condition and their families and caregivers. If you have NPC or care for someone with NPC, we invite you to join our research study and share your experiences. Your participation is crucial in helping us better understand NPC and improve support for those affected.

Learn more and find out how to get involved. Your voice matters!

Courageous Parents Network Virtual Workshop

Courageous Parents Network invites you to join their upcoming virtual workshop: Hear from the Siblings: Sharing Their Journey with Medical Complexity on Wednesday, April 23rd at 8:00 pm ET.

Do you wonder or worry about the siblings? Come hear from the siblings themselves about how loving and caring for a brother or sister with medical complexity shapes them. Join a panel of siblings to gain insights into the fears and worries that siblings carry, the things that helped them (and those that didn’t), and how loving a brother or sister with medical complexity shapes them.

This session will be moderated by Dr. Wendy Plante, who for over twenty years has worked with siblings of children with illness and disability and their families and has been involved with research and training to address siblings’ needs. Register here!

Be Part of the Story: Submit Your Photos!

We’d love to feature the incredible strength and spirit of our community by including your photos in our communications and promotions throughout the year! Whether it’s a family photo or a picture of your Niemann-Pick warrior, your photo helps tell our story. By sharing them, you’ll help us spread awareness, build support, and highlight the power of our amazing community. Please send your favorite photos today to [email protected]!

Thank you for supporting our NNPDF community. We truly appreciate and THANK each and every one of you for all you do.

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please contact Laurie Turner, Family Services Manager at 920-542-4038 or [email protected] if you have any questions about this program.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to host and support NPD fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

THANK YOU to those who have recently held Facebook Fundraisers!

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

NNPDF Store

Did you know our store has some great items to help raise awareness for Niemann-Pick Disease? Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options – available in T-Shirt or Hoodies. Personalization for your family is available. Be sure to check out our NNPDF apparel and logo items and send us your photos with your new gear! Allow 2 weeks for delivery. US only.

ASMD Accelerate

The Assistance Fund

Struggling with out-of-pocket expenses due to acid sphingomyelinase deficiency (ASMD)? The Assistance Fund is an independent charitable patient assistance organization helping patients and families by providing financial assistance for the cost of treatment and other health-related services. Through its Acid Sphingomyelinase Deficiency Financial Assistance Program, eligible individuals can receive assistance for all FDA-approved treatment for ASMD. Learn more.

Comprehensive Care Centers

NNPDF provides a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. Click here to learn more!

If you are connected with a medical team that would like to be added to the NNPDF Comprehensive Care Centers listing, please contact Laurie Turner at [email protected].

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact Laurie at [email protected] or call 920-542-4038

Supporting one another. Supporting our community.

March 2025 Newsletter

Newsletters

Message from our Executive Director

Dear Members of the NNPDF Community,

It is an honor to be joining this community of patients and families, and I look forward to working tirelessly on your behalf. With more than a decade of experience in the rare disease world and over 30 years in health care policy, my goal is to listen and learn from each of you as I work alongside the NNPDF Board to meet and exceed your expectations.

A little about me – after graduating from University of Virginia’s School of Law, I started my career on Capitol Hill in Washington, D.C., working in the House of Representatives and then the U.S. Senate. I then led reimbursement policy for Medtronic, a Minnesota medical device company before leading all U.S. policy, government affairs, and advocacy for an association representing cancer centers. Later, I led similar functions when I opened the Washington D.C. office for Sanofi, and then went on to lead Pfizer’s executive branch relationships, including FDA, CMS, and HHS. I have run my own consulting practice this past decade, where I have represented industry, patient, and provider organizations, successfully changing numerous policies to improve the environment for innovation and access to treatments for patients. I hope to bring this experience to NNPDF in ways that sustain and improve the services you have come to expect from NNPDF.

I live in the Washington, D.C. area – and perhaps, what is most important to know about me, I’ve been happily married for just over 25 years, with two wonderful children: one finishing college in Hoboken, NJ and one finishing graduate school in Australia. I’m also fortunate to live near my mom, also an attorney and my role model for how to have a fulfilling career and family.

I appreciate I have big shoes to fill, and I certainly can’t do this alone. While NNPDF has a wonderful Staff and Board, I look forward to hearing from each of you about your concerns and hopes for this organization and community. In the coming months, I will be reaching out proactively to as many of you as I can – and will find some time in the July Family Support & Medical Conference schedule as well – to learn from you. I also invite you to reach out to me at [email protected]. Let’s share in the commitment I have made to support you and NNPDF’s mission.

Warm regards,

Saira Sultan, JD
NNPDF Executive Director

July 10-13, 2025

Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa
Concord, North Carolina

We look forward to seeing you at our 33rd Annual NNPDF Family Support & Medical Conference! NNPDF has many exciting opportunities to connect with expert clinicians, researchers, and other Niemann-Pick families who share your journey. Be part of meaningful discussions, engaging family break-out sessions, important advocacy and FDA updates, Family Advisory Working Groups, our heartfelt We Remember Them video tribute, and much more.

Be sure to watch NNPDF communications for important updates. We look forward to seeing you there!

For all conference information visit nnpdf.org/conf.

Conference Registration

Early Bird Registration: Save by May 14th

Discounted Registration Rate:
Register by May 14th to take advantage of discounted registration fees.

Family Registration Discounted Rate:
EARLY BIRD REGISTRATION ONLY – Register 2 adults and 2 children (age 17 and younger) at the early bird registration rate of $500 (adults $175 and children $75). Any additional children in your immediate family will be free of charge.

First Time Attendee Discount:
Enjoy a 10% discount off your registration! We’re happy to have you join us!

Accommodations

Reserve by June 11th

Our 2025 conference will be held at the beautiful Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa in Concord, North Carolina. The NNPDF group rate will be available until June 11th, 2025. NNPDF Group Rate is $179 ($203.61 including taxes) per night. The NNPDF hotel group rate is available three days before and three days after the conference, dependent on hotel availability.

Family Conference Scholarships

Application Deadline April 10th

Scholarship funding is available to help NNPDF families ease the cost of attending the 2025 NNPDF Family Support and Medical Conference in Concord, North Carolina. Scholarship assistance will provide up to 3 nights hotel (room and tax only at the group rate) and up to 2 adult conference registrations for eligible applicants. Scholarship assistance is limited to immediate NNPDF U.S. family members of an individual with Niemann-Pick Disease.

NNPDF Family Advisory Working Groups

Application Deadline April 15th

The National Niemann-Pick Disease Foundation, Inc. (NNPDF) will host Family Advisory Working Groups that will take place on on Thursday, July 10, 2025 prior to the start of our Family Support & Medical Conference at the Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa in Concord, North Carolina.

Family Advisory Working Groups facilitate communication between Niemann-Pick families and the pharmaceutical industry, offering valuable insights into the patient and family experience. These sessions provide industry partners with the opportunity to engage privately with a small group of ASMD or NPC patients and/or immediate family members for a 3-hour discussion. This is an optional event and pre-registration is required.

NNPDF Cora Sterling Endurance Award

Application Deadline May 15th

Do you know an inspiring young person who’s making a difference in raising Niemann-Pick awareness? Nominate them for the NNPDF Cora Sterling Endurance Award! This special award is presented annually at the NNPDF Family Support & Medical Conference to a remarkable individual under age 25 who shines in raising awareness of Niemann-Pick disease in the community. Don’t miss the chance to celebrate their efforts!

Joele Ruppert & Joseph Colton ASMD Scholarship

Application Deadline May 15th

NNPDF is accepting applications for the Joele Ruppert and Joseph Colton ASMD Scholarship. This scholarship provides one-time funding of $500 to individuals diagnosed with ASMD for studies in post-secondary (high school) degrees, including two-year colleges, four-year colleges, vocational schools, and other post-secondary institutions.

Volunteer Opportunities

Volunteers play a vital role in supporting our Niemann-Pick community by raising awareness and helping our family support organization thrive. How can you contribute to NNPDF? We are reaching out to our community to see how YOU can help! We would like to know if you’re interested in offering your skills and time to assist NNPDF. All forms of support are greatly appreciated, currently we are looking for individuals who may be interested in the following:

Board Members
Committees
Focus Groups & Patient Advisory Committees
Advocacy and Outreach
Other specialized skills you may have!

If you are interested in becoming a NNPDF volunteer, please send your resume or a letter of interest and let us know what you are passionate about that may be an asset to the Foundation. Please share your work experience, hobbies, or skills. For example: I like working with numbers, I love to coordinate fundraising events, I work for a company that offers volunteer opportunities etc. Please reply by MARCH 30th to [email protected] and we will follow up with you.

Thank you for supporting our NNPDF community. We truly appreciate and THANK each and every one of you for all you do.

Supporting One Another. Supporting Our Community.

Rare Disease Week on Capitol Hill

NNPDF was proud to have one of our community members attend Rare Disease Week on Capitol Hill in Washington, DC last month. Garland Alvey attended the weeklong event.

Rare Disease Week on Capitol Hill empowers and inspires hundreds of advocates each year. Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases, brings together hundreds of rare disease individuals, family members, caregivers, and advocates from across the country to make their voices heard by their Members of Congress. Participants are educated on policy proposals impacting the rare disease community and provide opportunities to advocate for policy changes directly to their Members of Congress.

Thank you to Garland for sharing your voice and advocating on behalf of our entire Niemann-Pick Community. Read Garland’s summary report.

Be Part of the Story: Submit Your Photos!

We’d love to feature the incredible strength and spirit of our community by including your photos in our communications and promotions throughout the year! Whether it’s a family photo or a picture of your Niemann-Pick warrior, your photo helps tell our story. By sharing them, you’ll help us spread awareness, build support, and highlight the power of our amazing community. Please send your favorite photos today to [email protected]!

Thank you for supporting our NNPDF community. We truly appreciate and THANK each and every one of you for all you do.

NPC Study Opportunity: Help us better understand the impact of living with NPC

Niemann-Pick type C (NPC) impacts the quality of life for both those living with the condition and their families and caregivers. If you have NPC or care for someone with NPC, we invite you to join our research study and share your experiences. Your participation is crucial in helping us better understand NPC and improve support for those affected.

Learn more and find out how to get involved. Your voice matters!

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please contact Laurie Turner, Family Services Manager at 920-542-4038 or [email protected] if you have any questions about this program.

#RAREis Scholarship Application Open

Living with a #RareDisease? Have a dream to pursue your education? Perhaps a $5,000 scholarship would help. The #RAREis Scholarship Fund is now accepting applications for 2025!

The #RAREis Scholarship Fund is available for any rare disease patient who is age 17+ and has a U.S. residency. Applicants must be enrolled in an accredited course(s) for Fall 2025 Semester. Therefore, it is not degree specific!

If financial burden has held you back before, now is the time to pursue those dreams! Learn more.

Comprehensive Care Centers

NNPDF provides a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. Click here to learn more!

If you are connected with a medical team that would like to be added to the NNPDF Comprehensive Care Centers listing, please contact Laurie Turner at [email protected].

NNPDF Store

Did you know our store has some great items to help raise awareness for Niemann-Pick Disease? Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options – available in T-Shirt or Hoodies. Personalization for your family is available. Be sure to check out our NNPDF apparel and logo items and send us your photos with your new gear! Allow 2 weeks for delivery. US only.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to host and support NPD fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

THANK YOU to Pattie Hopkins, Lynn Ell, and Jennifer Walker Davis who recently held Facebook Fundraisers!

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

ASMD Accelerate

Clinical Trial Updates

Clinical trials are currently in progress to study and develop treatments for ASMD and NPC. The NNPDF posts new information regarding clinical trial updates as soon as it is received. Visit our Clinical Trials web page for up to date information on all clinical trials.

The Assistance Fund

Struggling with out-of-pocket expenses due to acid sphingomyelinase deficiency (ASMD)? The Assistance Fund is an independent charitable patient assistance organization helping patients and families by providing financial assistance for the cost of treatment and other health-related services. Through its Acid Sphingomyelinase Deficiency Financial Assistance Program, eligible individuals can receive assistance for all FDA-approved treatment for ASMD. Learn more.

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact Laurie at [email protected] or call 920-542-4038

Category: Newsletters

Newsletters

A Message to Our Community

Rare Disease Day is February 28th!​

Kasey Apt

Mom of Tinley, ASMD

Tell us a bit about you and your family…

Sharing Your Family’s Diagnosis Journey

Continuing the Conversation

International Niemann-Pick Disease Registry

YARR Leadership Academy

Family Assistance & Support Program

Fundraising

ASMD and NPC Care Clinics

Plain Language Summaries

Milestones

Get Your NNPDF Gear and Awareness Apparel for Rare Disease Day

NNPDF Membership

With gratitude to our Funding Partners

We are grateful to these companies for their trust and support in advancing our mission.

Supporting one another. Supporting our community.

Newsletters

A Message to Our Community

Jesika Coulbern

Mom of Kayden, NPC

Tell us a bit about you and your family…

Rare Disease Day is February 28th!​

NNPDF Member Survey

Bringing Holiday Cheer Update

Family Assistance & Support Program

Surveys, Studies & Market Research

NPC Patient Services Market Research Study

NPC Brain Imaging Research Study for NPC

Community News Updates

Fundraising

ASMD and NPC Care Clinics

Plain Language Summaries

Milestones

Get Your NNPDF Gear and Awareness Apparel for Rare Disease Day

NNPDF Membership

With gratitude to our Funding Partners

We are grateful to these companies for their trust and support in advancing our mission.

Supporting one another. Supporting our community.

Newsletters

A Message from our Board Chair

We Remember Them

Milestones

Plain Language Summaries

ASMD and NPC Care Clinics

Coming Soon! Tools for Meaningful Conversations

Family Assistance & Support Program

Community News Updates

Fundraising

Surveys, Studies & Market Research

NPC Brain Imaging Research Study for NPC

NNPDF Gear and Awareness Apparel

NNPDF Membership

With gratitude to our Funding Partners

We are grateful to these companies for their trust and support in advancing our mission.

Supporting one another. Supporting our community.

Newsletters

A Message from our Board Chair

Bringing Holiday Cheer

Application deadline is Sunday, November 30

Celebrating #GivingTuesday

The Assistance Fund 2026 Reenrollment

Family Assistance & Support Program

Milestones

Fundraising Events

Pampered Chef Fundraiser for NNPDF

Smoothie King Fundraiser for NNPDF

19th Annual NNPDF Dillon Papier Baysox Benefit

Surveys, Studies & Market Research

NPC Brain Imaging Research Study for NPC

Clinical Trial Updates

NNPDF Gear and Awareness Apparel

Comprehensive Care Centers

NNPDF Membership

OUR VALUED PARTNERS

Supporting one another. Supporting our community.

Rare Disease Day is February 28th!

Rare Disease Day is February 28th!