Wednesday, 9/10/25 – In advance of the 2025 American Neurological Association (ANA) meeting, Mandos Health releases two scientific abstracts with promising data for the treatment of NPC patients with Adrabetadex. The lead abstract, designated by ANA as an “Abstract of Distinction,” presents data showing a statistically significant survival benefit in patients with infantile-onset NPC who …
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Niemann-Pick Type C News
Zevra Therapeutics to Sell Priority-Review Voucher for $150 Million related to NPC drug approval
February 27, 2025 at 7:31 AM EST CELEBRATION, Fla., Feb. 27, 2025 (GLOBE NEWSWIRE) — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease …
7th Annual DART to the Finish Charity Walk Raises Over $160,000
GREENWICH, Conn., September 30, 2024 – Dana’s Angels Research Trust (DART), the nonprofit organization dedicated to funding medical research, education and treatment of the rare childhood disease Niemann-Pick Type C (NPC), often referred to as “childhood Alzheimer’s,” is proud to announce that its 7th annual DART to the Finish Charity Walk, held on September 28, …