November 2025 Newsletter

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]

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