NNPDF

NNPDF is sharing an opportunity from Beren Therapeutics for NPC patients and caregivers to participate in a 60-minute virtual interview focused on disease awareness and early diagnosis. learn more at ow.ly/nBqt50Yx35m

Beren Therapeutics announced that the U.S. FDA has accepted its NDA for adrabetadex in infantile-onset Niemann-Pick type C (NPC) with Priority Review. PDUFA date: August 17, 2026. This acceptance is an important milestone for the NPC community. Read more: ow.ly/4pPR50YofIm

Our February NNPDF Newsletter is here! Stay up to date with the latest community news, Rare Disease Day information, Community Voices Spotlight, and important foundation updates. Read the full newsletter and stay connected at nnpdf.org/february-2026-…

NNPDF is seeking a compassionate, caring Family Services Manager to join our team in supporting the needs of families affected by Niemann‑Pick disease and strengthening the programs and services that support our community. Learn more at t.ly/NNPDF-fsm2026

Thank you to our dedicated Scientific Advisory Board for your unwavering commitment and expertise. Your work impacts the Niemann-Pick community and advances care and hope for families affected by rare diseases. We are truly grateful for all you do! 💜 #RareDiseaseDay #NiemannPick

NNPDF is seeking an experienced and passionate leader to serve as its Executive Director - to lead a small, capable, and dedicated team that is making a meaningful difference for individuals and families affected by Niemann-Pick disease. Learn more at t.ly/NNPDF-ed2026

#RareDiseaseDay is a time to shine a light on Niemann-Pick Disease, along with the millions affected with rare diseases worldwide. Every share can raise awareness, bring hope, and inspire change! GET INVOLVED at nnpdf.org/rdd/. #NiemannPick #ASMD #NPC

Our January 2026 Newsletter has just been published! Check out our Community Voices Spotlight featuring Jesika Coulbern, community updates, upcoming Rare Disease Day information, and more at nnpdf.org/january-2026-n…

We’re Still Listening! Take a moment to complete our NNPDF Member Survey! Share your thoughts to help NNPDF provide the best possible support and resources for you and your family. Visit ow.ly/eaRX50Y1Y72

IMPORTANT: Our previous Member Survey link had a technical error. If you’ve already filled it out, please re-submit your responses. We apologize for the inconvenience and truly appreciate your time! NNEW SURVEY LINK: t.ly/c4sGC

Stay connected with the NNPDF December newsletter! This issue features a message from our Board Chair, new Plain Language Summaries of research, a special milestone celebration, and details for our upcoming webinar. Read more at t.ly/Un4f6

Join NNPDF on Jan 8, 2026, at 8 PM ET for a webinar featuring Mandos/Beren Therapeutics providing information on the recent adrabetadex NDA filing and Early Access Program. See announcement at t.ly/0zl6J Pre-register for the webinar at: t.ly/PFPXH

NNPDF is pleased to share that Mandos Health has received Breakthrough Therapy Designation (BTD) from the U.S. FDA for adrabetadex, an investigational drug for infantile-onset NPC. Read the complete announcement at ow.ly/xmtq50XH7zm

NNPDF is sharing an opportunity for NPC patients and caregivers to take part in market research sponsored by Mandos Health (Beren Therapeutics) to better understand the needs and experiences of those using current NPC treatments. Learn more at t.ly/KC-w8

Warm wishes for a wonderful Thanksgiving!

Welcome to our November update! Catch up on community events, an update from the Board Chair, a Bringing Holiday Cheer reminder, and more! Read more at nnpdf.org/november-2025-…

NNPDF is pleased to announce the first U.S. study site for Azafaros' Phase 3 NAVIGATE clinical trial. This study evaluates the safety and efficacy of nizubaglustat for treating late-infantile/juvenile NPC and GM1/GM2 diseases. Read more at #11.19.25" target="_blank" rel="nofollow noopener">nnpdf.org/azafaros/#11.1…

Tomorrow’s #OpenEnrollment "Stay Covered" webinar at 2pm ET helps you understand changes to Medicare, Medicaid, and the ACA and how they affect access to affordable health care for rare disease families. Register here: bit.ly/4nUYIEm

When you give, you’re helping build a stronger, more supportive community for families facing Niemann-Pick. Learn more and join us in making a difference at nnpdf.org. Supporting One Another. Supporting Our Community. #NiemannPickAwareness #AMSD #NPC

Families impacted by Niemann-Pick don’t have to face it alone. NNPDF provides programs, resources, and a caring network to help every step of the way. Learn more at nnpdf.org. Supporting One Another. Supporting Our Community. #NiemannPickAwareness