NNPDF

We’re extending Early Bird Registration for the NNPDF Conference to May 21st! Join us for a weekend of education, connection, and hope - don’t miss the Early Bird rate! Registration information: nnpdf.org/conf-register/

Just a friendly reminder to register for the Family Support & Medical Conference by May 17th to take advantage of the Early Bird registration rates! We look forward to an incredible time together and can’t wait to see you all in Orlando! Learn more at nnpdf.org/conf/

The NNPDF is happy to share our April 2026 News & Updates! Check it out at nnpdf.org/april-2026-new…

We are thrilled to share incredible news! Acid Sphingomyelinase Deficiency (ASMD) has officially been approved for inclusion on Wisconsin’s Newborn Screening Panel. Read complete announcement at #WI2026" target="_blank" rel="nofollow noopener">nnpdf.org/newborn-screen…

UPDATE FROM AZAFAROS NNPDF is pleased to share that additional U.S. study sites for Azafaros' Phase 3 NAVIGATE study will be opening in the coming weeks. Read the complete update and register for a Community Update Webinar with Azafaros at ow.ly/b04450YLxEc

Join us for Community Connections Chat on Wednesday, April 29 at 6:00pm ET for an informal open space to share, ask questions, and connect with others in our Niemann-Pick community. Join the conversation! Register at t.ly/ZW3co

We’re excited to share the NNPDF’s March 2026 News & Updates! Take a look and see what’s new! nnpdf.org/march-2026-new…

Join us for our Family Support & Medical Conference! Connect with families, caregivers, clinicians, researchers, advocates, funding partners, and our Board of Directors and Scientific Advisory Board to share, support, and celebrate our community. Visit nnpdf.org/conf/

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