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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Azafaros Secures €132M in Financing to advance Phase 3 clinical programs, including NPC
13 May 2025 Leiden, Netherlands, 13 May 2025 – Azafaros, a clinical-stage company focused on developing disease-modifying therapeutics to offer new treatment options to patients with rare lysosomal storage disorders, announces the completion of an oversubscribed €132M Series B financing led by Jeito Capital, co-led by Forbion Growth and with additional participation from Seroba, Pictet Group …
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 …