A Message from the Executive Director
Dear Friends,
In just 14 days, we will come together for our Annual Family Support & Medical Conference, and I could not be more excited. This conference is one of the highlights of our year, a time when families, caregivers, advocates, clinicians, and community partners gather to learn, connect, and strengthen one another. While the educational sessions and resources are invaluable, it is the relationships and shared experiences that make this event truly special. Our team has been working tirelessly... READ MORE
| | Just two weeks to go! Online registration closes July 3, so reserve your spot today. Registrations received after July 3 must be completed in person. | | |
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 Allie Stavrolakis at 877-287-3672 or email familyservices@nnpdf.org if you have any questions about this program.
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Update from Beren Therapeutics
BerenTherapeutics has shared a community update outlining its long-term commitment to the NPC community.
Building on its previously announced $300 million financing commitment and newly secured long-term funding, the company plans to expand programs that support families throughout the diagnosis and ongoing care journey.
Read the complete update and learn more about the resources being developed.
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Rare Artist Contest Celebrates Creativity in the Rare Disease Community
We’re excited to share the Rare Artist contest from the EveryLife Foundation for Rare Diseases, celebrating the creativity of those impacted by rare diseases. This year’s contest features music, poetry, visual art, and a new short-form video category... READ MORE
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CALIFORNIA ADVOCATES
California is encouraging patient advocates to testify in support of AB 1887 (bill to end prior authorization and step therapy for rare disease patients) at the Senate Health Committee hearing in Sacramento on July 1 at 12:00pm PT. Please reach out to your California constituents so we can pass this bill to end these insurance practices.
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GEORGIA RESIDENTS: TAKE ACTION FOR NEWBORN SCREENING
Georgia is looking for families to reach out to legislators in support of funding of the newborn screening pilot program.
In January of 2025, the Georgia Newborn Screening and Genetic Advisory Committee recommended a two-year pilot study adding (ASMD, Fabry, and Gaucher) to their newborn screening panel. To proceed with the pilot, funding needs to be included... READ MORE
| | Our Amazing Fundraising Supporters | | |
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 Tracie Chatwood, Jamie LeBouthillier, Allison Reiter, and Tracey Szuran 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!
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Update from Cyclo Therapeutics
Research progress in NPC continues with CycloTherapeutics announcing that the final participant has completed the 96-week visit in the Phase 3 TransportNPC™ study evaluating Trappsol® Cyclo™ for Niemann-Pick disease Type C. The study enrolled 94 patients across 27 sites in 13 countries and expects to announce top-line results and pursue an NDA filing later this year.
Cyclo is thankful to the families, clinicians, researchers, and advocates whose participation and commitment help advance NPC research.
Read the complete update.
| 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. | |
Click here to update or enroll today!
For assistance contact us at nnpdf@nnpdf.org
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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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