March 2026 Newsletter

A Message to Our Community

Dear Friends,

We are energized by the opportunities ahead to connect, learn, and grow together as a community. One of the highlights we are especially looking forward to is our 34th Annual NNPDF Family Support & Medical Conference.

Registration is now open, and we warmly invite you to join us for this meaningful and inspiring gathering. This conference is a special time for families, caregivers, clinicians, researchers, and advocates to come together, sharing experiences, building connections, and strengthening the support that defines our community.

We are actively working to shape a thoughtful and engaging agenda that reflects the needs and voices of our community. Each year, this event reminds us of the power of connection and the importance of coming together to support one another.

We hope you will join us and be part of this incredible experience.

Respectfully,
The NNPDF Team

Volunteer Opportunities

Volunteers are an essential part of our Niemann-Pick community, helping raise awareness and supporting our family organization’s work.

We’re reaching out to see how you might like to get involved. Whether you can share a little time or bring a special skill, your help makes a real difference for families living with Niemann-Pick. All forms of support are greatly appreciated. Currently, we are looking for individuals who may be interested in the following areas:

Advocacy and Outreach
Board Members
Committee Members for:
- Family Services
- Communications and Marketing
Family Conference and Regional Family Events
Other specialized skills you may have!

If you are interested in volunteering with NNPDF, please send a resume or brief letter of interest to [email protected]. Let us know what you are passionate about and how your skills or experience could support the Foundation, including work background, hobbies, or other talents. For example, you might enjoy working with numbers, coordinating events, or participating in volunteer programs through your company.

Thank you for supporting the NNPDF community. We truly appreciate all that you do for families affected by Niemann-Pick disease.

Rare Disease Week on Capitol Hill

We were honored to have community member Christine Petty and her son Ryan represent NNPDF at Rare Disease Week on Capitol Hill in Washington, DC last month. Christine and Ryan participated in the weeklong event, joining advocates from across the country.

Rare Disease Week on Capitol Hill is an inspiring annual event that brings together individuals living with rare diseases, along with their families, caregivers, and supporters. Organized by Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases, the event provides participants with important updates on policy issues affecting the rare disease community and equips them with the tools to engage directly with Members of Congress.

We are grateful to Christine for lending her voice and advocating on behalf of the Niemann-Pick community. Be sure to read Christine’s summary report to learn more about her experience.

Michael and Jeannine Hill

Parents of Morgan, NPC

Tell Us a Little About You and Your Family…

Hi! We are Michael and Jeannine Hill, parents of three amazing “super siblings” – Travis (31), Jenna (27), Spencer (23), and one incredibly resilient NPC warrior, Morgan (20). Our home is located in West Point, Utah. We have three very busy dogs, which always keep our house full of life: a Bernedoodle, a Shichon, and a Brittany Spaniel. Morgan’s diagnosis of Niemann-Pick disease type C has definitely shaped our journey as a family. While it has brought many challenges, it has also drawn our family closer together, reminding us to cherish every moment.

Sharing Your Family’s Diagnosis Journey

When Morgan was 16 years old, we began to notice that something wasn’t quite right, because she would stumble quite often while playing with the dogs and other daily activities were getting harder for her to do. It was small at first, but as it continued to progress, her hands would curl inwards while walking, and other symptoms were starting to manifest, and we were growing more concerned. We then spoke with her primary care physician, who referred us to a neurologist.

What followed was a long series of tests, and with each one we were hoping would explain the cause of her symptoms of ataxia, and each one coming back as normal. Still with no answers and increasing uncertainty… CONTINUE READING

Courageous Parents Network Virtual Workshop

Courageous Parents Network invites you to join their upcoming virtual workshop: Building Bridges, Breaking Down Walls: Transforming Conflict in the Care of Medically Complex Children on Monday April 13, 2026 at 8:00 pm ET.

Conflict often arises when caring for medically complex children due to differences in values, communication, and perspectives. This session explores what drives conflict and offers practical ways to navigate it while preserving relationships and focusing on what matters most. Join us to learn how conflict escalates, how to de-escalate it, and how to approach difficult conversations with greater understanding and intention. Learn more and register.

Family Caregiver Alliance
Family Caregiver Alliance supports caregivers of adults with chronic or disabling conditions, including those living with Niemann-Pick disease who are over 18. They provide practical resources, education, training, and emotional support to help families manage daily challenges and improve quality of life. The organization also advocates for caregiver needs through research, policy, and awareness initiatives nationwide. Learn more.

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

NORD’s ASMD Patient Assistance Programs

NORD has offered assistance programs since 1987 to help patients access critical, life-sustaining medications and other support that might otherwise be out of reach. For individuals in the ASMD community, NORD’s Patient Assistance Programs may also help cover insurance premiums, co-pays, diagnostic testing, and even travel related to clinical trials or specialized care. Learn more.

Milestones

Aaron and Kelly Schoenecker traded Minnesota’s chilly winds for the sparkling turquoise waters of Grand Cayman! With sun, sand, and a little kid-free relaxation, they made unforgettable memories and had the getaway of a lifetime!

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!

Books & Publications Highlight

A Ribbon for Your Hair is a story about Stephen Policoff’s journey as he cares for his adopted daughter Anna, diagnosed with Niemann-Pick Type C disease, while also facing his wife Kate’s late-stage cancer. The memoir weaves together heartbreak, dark humor, and deep tenderness, celebrating resilience, love, and the small joys that endure even in the face of tragedy.

This book is part of a collection of works by Niemann-Pick family authors available on our Books & Publications page.

Surveys, Studies & Market Research

NPC Disease Awareness – Concept Market Research

NNPDF invites the NPC community to share their feedback on NPC disease awareness materials in a market research project sponsored by Beren Therapeutics.

The purpose of this market research is to gather your feedback on messaging, images, and communication tools that effectively support the early diagnosis of NPC, as well as your insights on genetic testing offerings and referrals to NPC Sites of Care. 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.

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 Update

Update from Beren Therapeutics
Beren Therapeutics is please to announce that the U.S. Food and Drug Administration has accepted its New Drug Application for adrabetadex in infantile-onset Niemann-Pick disease type C (NPC) and granted Priority Review, with a PDUFA date of August 17, 2026. This acceptance is an important milestone, reflecting the significant unmet need in infantile-onset NPC and incorporating feedback from the FDA and the NPC community. Read the complete press release.

Get Your 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.

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 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]