NPC is an ultra-rare, relentlessly progressive, genetic disorder that leads to premature mortality. MIPLYFFA, used in conjunction with miglustat, is the only treatment shown to halt disease ...
Dublin, Aug. 01, 2025 (GLOBE NEWSWIRE) -- The "Niemann-Pick Disease Type C NPC Market - A Global and Regional Analysis: Focus on Country and Region - Analysis and Forecast, 2025-2035" report has been ...
Beren Therapeutics P.B.C. subsidiary Mandos LLC submitted a NDA to the FDA for adrabetadex for the treatment of infantile-onset NPC.
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