National Niemann-Pick Disease Foundation
Joined February 2009
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Beren Therapeutics has shared an update outlining its commitment to the NPC community with plans to expand programs supporting families throughout the diagnosis and care journey. Review the full update at bit.ly/4v2KBkZ
#NiemannPick #NPC #RareDisease #ClinicalResearch
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NPC research progress continues. CycloTherapeutics has announced that the final participant has completed the 96-week visit in the Phase 3 TransportNPC™ study evaluating Trappsol® Cyclo™ for NPC. Read more at ow.ly/UC0P50ZagT4
#NPC #RareDisease #RareDiseaseResearch
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📣 Good News, NNPDF Community!
We've extended the deadline to secure the discounted conference hotel room rate for the 2026 NNPDF Family Support & Medical Conference. Visit nnpdf.org/accommodations/ today!
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📣 Your Story Matters
Zevra Therapeutics is seeking NPC patients, family members, and caregivers to share their stories. Your voice can help families feel less alone and raise awareness of life with NPC. Learn more: t.ly/QOiPq
#NPC #RareDisease #PatientVoice
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🎉 We can't wait to see you in Orlando!
NNPDF Conference discount deadlines are approaching:
✅ Hotel group rate ends June 8
✅ Register by June 8 to receive your conference shirt
✅ Free conference shipping on swag
Learn more at conta.cc/4e2PO4K
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📣 NNPDF is excited to welcome Heather Moran-Teal as it's new Executive Director. 💙 Her passion for rare disease advocacy will help lead next chapter of support, education, and hope. Read more at t.ly/gCWTz #NNPDF #NiemannPickDisease #RareDisease #PatientAdvocacy
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Check out our Conference Agenda at a Glance! 💙 Reconnect with the NNPDF family, meet new faces, learn, collaborate, and strengthen our community together. Register book your room today: nnpdf.org/conf
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In our latest News & Updates edition, we welcome Heather Teal, NNPDF Executive Director, share important conference updates, spotlight the Peterson family, celebrate milestones, and more! Read it here: nnpdf.org/may-2026-news/
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CONFERENCE ATTENDEES: Order your NNPDF gear by June 9 for free shipping to the Family Support & Medical Conference! Shop now at nnpdf.org/store/
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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/
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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/
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The NNPDF is happy to share our April 2026 News & Updates! Check it out at nnpdf.org/april-2026-newslet…
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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 nnpdf.org/newborn-screening/…
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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
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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
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We’re excited to share the NNPDF’s March 2026 News & Updates! Take a look and see what’s new! nnpdf.org/march-2026-newslet…
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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/
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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
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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
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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-news…
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