January 2026 Newsletter

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]

January 2026 Newsletter