Zevra Therapeutics Inc (ZVRA) marks a successful transition to a commercial stage with MyPIFA's launch, despite financial ...

ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...

The PRV was granted to Zevra in September 2024 following approval by the U.S. Food and Drug Administration of MIPLYFFA (arimoclomol), which is indicated for use in combination with miglustat for ...

The PRV was granted to Zevra in September 2024 following approval by the U.S. Food and Drug Administration of MIPLYFFA (arimoclomol), which is indicated for use in combination with miglustat for the ...

This study reports that activation of TFEB promotes lysosomal exocytosis and clearance of cholesterol from lysosomes, the strength of evidence for which is convincing with appropriate and validated ...

MIPLYFFA is approved for use alongside miglustat in patients aged two and older with neurological manifestations of NPC. Zevra's Chief Medical Officer, Adrian Quartel, highlighted the FDA approval ...

Collaborative Innovation Center of Yangtze River Delta Region Green Pharmaceuticals, College of Pharmaceutical Sciences, Zhejiang University of Technology, Hangzhou, China Department of Molecular, ...