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Dear Friends,
It has been a busy and meaningful fall! From the World.Fair Lysosomal Storage Disease meeting in September to the Global Genes Rare Drug Development Symposium in Boston, a NNPDF regional event in Chicago, and now the NORD Summit in Washington, D.C., I am grateful for the opportunity to connect, learn, and represent our Niemann-Pick community.
There is something special about coming together with the greater rare disease community to share our successes, challenges, and ideas. One message continues to resonate across these conversations: delays in diagnosis remain a shared challenge across rare diseases, and time matters. Identifying a disease in the first days of life, rather than months or even years later following a long diagnostic odyssey, can make a critical difference, particularly when irreversible damage may begin before symptoms appear.
As we wrap up Niemann-Pick Awareness Month, thank you to everyone who helped shine a light on this disease. Your stories, voices, and willingness to share your experiences help raise awareness and remind us why early identification, timely care, and continued advocacy are so important.
I remain in awe of the families I have the privilege of serving. Please know that I carry your voices into these conversations and remain deeply committed to pushing forward at every level to ensure our community is heard, supported, and never forgotten.
With heartfelt gratitude,
| | NNPDF Family Toolkit & Website Launch​ | | |
Join us Wednesday, October 28 at 6:00 p.m. EST for a special evening as we celebrate the LIVE launch of our new Family Toolkit and brand-new website!
Join NNPDF Chair Taylor Sabky and Vice Chair Kelly Lee as they share the “why” behind the Family Toolkit, and how this important new resource will help support and empower our community.
They'll be joined by special guest speakers Jonathan Schorr, Founding Partner, Leslie Cory, Director, Strategic Communications, and Fallon Moore, Associate, Strategic Communications from BeClear, who will share how this vision came to life.
We'll take you on a live tour of the newly redesigned NNPDF website and demonstrate the Family Toolkit, showing how families can access information, navigate resources, and find support. This is more than a website launch, it’s an exciting new chapter in how we connect, inform, and empower our community. We can't wait to share it with you!
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West Coast Regional Family Event -
Now Offering Virtual Participation!
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Good news! Our West Coast Regional Family Event on November 7 is now hybrid, offering families the option to participate virtually or attend in person.
Join NNPDF for a day of connection, learning, and support with opportunities to connect with other Niemann-Pick families, hear from clinicians and NNPDF staff, learn about research and treatment updates, and explore whole-person wellness.
For those joining us in person, we’ll kick things off Friday evening with a pizza social. 🍕
Whether you join us in person or virtually, we look forward to bringing our community together!
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Finding Connection, Support, and Yourself Along the Caregiving Journey
In recognition of National Family Caregivers Month, we invite family caregivers to join us for a special virtual Caregiver Support Summit.

Date: November 17
Time: 7:30 - 9:00 PM ET
Location: Virtual
Caregiving can change every part of life, from family relationships and daily responsibilities to how we see ourselves. While every caregiving journey is different, no one should have to navigate its challenges alone.
This interactive event will bring caregivers together for candid conversations, practical guidance, and meaningful connection. Hear directly from family caregivers about their experiences, explore how caregiving affects the entire family, and discover ways to stay connected to your own identity and wellbeing while caring for someone you love.
Attendees will also have the opportunity to connect with others in small, facilitated breakout groups based on their caregiving roles, share experiences, ask questions, and leave with practical ideas and resources they can use in everyday life.
Whether you're a parent, spouse, sibling, or adult child providing care, this summit is a space for you to feel heard, understood, and supported.
This webinar was made possible through an educational grant by Zevra Therapeutics.
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NNPDF is encouraged by promising Phase 3 TransportNPC™ findings for Trappsol® Cyclo™, including slower disease progression and encouraging survival outcomes in infantile-onset NPC. These findings reinforce the urgent need for newborn screening, earlier diagnosis, and treatment options for children affected by NPC. Cyclo Therapeutics anticipates submitting an FDA New Drug Application in Q4 2026.
Read the full press release.
SEPTEMBER 30,2026
| | Beren Therapeutics Presents New Adrabetadex Data | | |
New research offers encouraging news for the Niemann-Pick disease type C (NPC) community. Beren Therapeutics has announced new adrabetadex data showing preservation of neurological function for more than 10 years in children with infantile-onset NPC.
Read the full press release.
OCTOBER 15, 2026
| | Rare Disease Week​ Scholarship | | If you are interested in participating in Rare Disease Week and Rare Advocacy Week on Capitol Hill March 2-4, 2027, we encourage you to apply early for the scholarship opportunities being offered by the RARE Foundation. | | |
NNPDF will also be in Washington, DC during Rare Disease Week! We’re excited to use this opportunity to bring our community together on March 1, 2027 and are planning a regional family event to align with the week’s activities. 

A limited number of scholarships will also be available for families interested in attending the regional event to help support participation. More details coming soon! We hope to see many of our families there!
Deadline for the RARE Foundation scholarship application is Friday, November 6, 2026. Apply here.
| | | Rare Disease Day Kickoff Webinar | | |
In conjunction with Rare Disease Week, Rare Disease Day, recognized annually on the last day of February, unites millions worldwide to raise awareness and support the rare disease community.
Join NORD on November 18 at 4:00 p.m. ET for the 2027 Rare Disease Day Kickoff Webinar to celebrate the U.S. launch and discover ways to get involved. Register here.
| | | Niemann-Pick Disease Awareness Month | | |
Throughout October, we came together to recognize Niemann-Pick Disease Awareness Month and make Niemann-Pick disease more visible. We are incredibly grateful to our community, partner advocates, families, and supporters who participated by sharing their stories, photos, and experiences. Every post and shared moment helped raise awareness of ASMD and NPC, build understanding, and remind others that behind every diagnosis is a person, a family, and a community deserving of support.

Your voices make a difference, and together, we can continue sharing stories, strengthening connections, and keeping Niemann-Pick disease visible throughout the year.
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Family Assistance & Support Program
Family Assistance & Support Program
The Family Assistance & Support Program (FASP) offers short-term financial assistance to eligible NNPDF member families experiencing an unexpected financial hardship or crisis. Covered expenses may include specialized medical equipment and goods, utility bills, adaptive and other home repairs, car repairs and payments, rent, mortgage payments, and bereavement costs, among others.
Sometimes a little extra help can make all the difference. If you're facing an unexpected expense or need support during a difficult time, we're here for you. Learn more about the Family Assistance & Support Program and apply for assistance.

Contact Family Services Manager, Allie Stavrolakis at 877-287-3672 or email familyservices@nnpdf.org if you have any questions about this program.
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You don’t have to navigate this journey alone.
The journey with NPC or ASMD brings unique challenges, emotions, and uncertainties at every stage. NNPDF is here to support you and your family throughout the journey, offering guidance, resources, and compassionate support when you need it most.
Whether you have questions, need help navigating challenges, or simply need someone to talk to, our Family Services Manager, Allie Stavrolakis, is here for you. You and your family are not alone.
| | Your Participation Helps Advance Research | | Every voice matters. By participating in surveys and studies, individuals and families affected by Niemann-Pick disease share valuable insights that help guide research, support treatment development, improve services, and move our community closer to meaningful progress. | | National Institutes of Health Studies | | Invite your family, friends, and support network to become part of the NNPDF community. Membership is free and the best way to stay informed on the latest news, updates, and resources. Enrolling, confirming, or updating your membership helps ensure we have accurate information for you and your family so we can continue providing relevant information and updates. | | | |
NATIONAL NIEMANN-PICK
DISEASE FOUNDATION, INC.
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DISCLAIMER: The National Niemann-Pick Disease Foundation (NNPDF) does not engage in the practice of medicine. It is not a medical authority, nor does it claim to have medical knowledge. The NNPDF website is an educational service of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on our website does not constitute medical advice. For all information related to care, medication, or treatment, the NNPDF recommends consulting a physician to determine if information presented is applicable.
This communication is a service of the National Niemann-Pick Disease Foundation's Family Services Program, made possible by the financial support of our member families and other generous donors and grantors. The NNPDF receives no government funding, nor does it host or receive funding from advertising or from the display of commercial content.

The National Niemann-Pick Disease Foundation is a 501(c)(3) organization. Your donations are fully tax-deductible.
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