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 ...
Four years of real-world data from the U.S. EAP demonstrate that arimoclomol was well tolerated and stabilized disease progression in the overall cohort, with changes in clinical severity scores ...
A Zevra Therapeutics drug is now the first FDA-approved therapy for an ultra-rare, inherited metabolic disorder whose effects on the central nervous system can become fatal by the time a patient is a ...
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 ...
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