June 2026 Newsletter
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 behind the scenes to create an experience that is meaningful, impactful, and centered on community. We also have a few exciting surprises in store that we cannot wait to share with you.
On a personal note, this will be my first conference with this incredible community. Over the past several months, I have had the privilege of learning from many of you, hearing your stories, and witnessing the resilience, compassion, and dedication that make this community so remarkable. That is why I am especially looking forward to gathering together in person.
Most of all, I look forward to meeting each of you face-to-face. Whether this is your first conference or you have been part of our community for years, your presence matters. Together, we are building something bigger than a conference – we are strengthening a network of support, hope, and connection that extends far beyond a single weekend.
Thank you for being part of this journey. Safe travels, and I’ll see you soon.
With gratitude,
Heather Moran-Teal, M.Ed.
Executive Director
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.
I’m really looking forward to getting to know the Niemann-Pick community and meeting some of you in person next month in Orlando! I’m here to support you however I can, whether that’s providing information, connecting you with services, or sharing community resources. Please feel free to reach out anytime, whether you need assistance or just want to say hello. I’d love to connect! Read Allie’s bio.
Allie Stavrolakis
Family Services Manager
[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 contact Allie Stavrolakis at 877-287-3672 or email [email protected] if you have any questions about this program.
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.
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. The contest is open to anyone in the U.S. who has a rare disease or is connected to someone who does. Submissions close July 20, 2026. Learn more and enter.
Rare Artist is a national advocacy initiative at the intersection of art and health. By harnessing the power of creative expression, the community turns lived rare disease experiences into advocacy, storytelling, and opportunities for broader public awareness and impact.
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.
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 in the Georgia state budget.
Please email and call your state legislators and ask them to urge the Commissioner of Public Health to include $2 million in this year’s budget, as recommended by the Georgia Newborn Screening and Genetics Advisory Committee, or to allocate the funding during the budget process. Find your Representative and Senator.
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!
Clinical Trial Update
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.
Stay On Top of the Latest News
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 [email protected]
With gratitude to our Funding Partners
We are grateful to these companies for their trust and support in advancing our mission.
