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 ...
Niemann-Pick Disease Type C (NPC), an ultra-rare, progressive neurodegenerative disease, is heterogeneous and is often misdiagnosed or undiagnosed. A recent study aimed to get a fuller picture of the ...
CELEBRATION, Fla., Dec. 29, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with ...
LEIDEN, Netherlands--(BUSINESS WIRE)--Azafaros has announced that data from the ongoing double-blind, placebo-controlled Phase 2 RAINBOW study investigating its lead asset, nizubaglustat in patients ...
CENTOGENE and Insilico Medicine will work together to identify, rank, and annotate proteins to identify novel therapeutic targets for NPC using AI NPC is a rare genetic disease occurring in ...
Nizubaglustat is a small molecule, orally available and brain penetrant azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with ...
IntraBio’s Aqneursa receives European Commission’s marketing approval for treatment of Niemann-Pick Type C disease: Austin, Texas Saturday, January 24, 2026, 14:00 Hrs [IST] I ...
First patient dosed in global pivotal clinical trial program for late infantile/juvenile onset NPC and GM1/GM2 gangliosidoses. Both studies are part of Azafaros’ mission to develop treatments for the ...
IntraBio Inc. today announced that the European Commission granted marketing authorization to AQNEURSA(levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick Type C (NPC) ...
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