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Washington, D.C., Feb. 03, 2026 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today applauded the bipartisan passage of the annual federal funding bill that sustains critical...

Exploratory Phase 2a study showed consistent and clinically meaningful functional improvements across muscle strength, motor performance, and patient-reported outcomes in patients with...

Exploratory Phase 2a study showed consistent and clinically meaningful functional improvements across muscle strength, motor performance, and patient-reported outcomes in patients with...

Le venglustat de Sanofi a atteint tous les critères d'évaluation principaux dans une étude de phase 3 sur la maladie de Gaucher de type 3 Dans l’étude de phase 3 LEAP2MONO, le venglustat, administré...

Sanofi's investigational drug venglustat improved the neurologic symptoms of type 3 Gaucher disease, for which there are currently no approved therapies.

CHICAGO, Jan. 29, 2026 (GLOBE NEWSWIRE) -- Quetzal Therapeutics, a biopharmaceutical company focused on developing treatments for rare diseases, announced that the U.S. Food and Drug Administration...

Dublin, Jan. 29, 2026 (GLOBE NEWSWIRE) -- The "Nucleic Acid Therapeutics Contract Manufacturing Market Size, Share & Trends Analysis Report by Type (Gene Therapy, RNA-based Therapies), Service,...

Dublin, Jan. 29, 2026 (GLOBE NEWSWIRE) -- The "Healthcare Contract Sales Organizations Market Size, Share & Trends Analysis Report by Service (Personal Promotion), Therapeutic Area, End Use and...

Not intended for the UK Apecotrep (BI 764198) is a potential first-in-class, oral, selective TRPC6 inhibitor being investigated as a novel, targeted, non-immunosuppressive therapy in people with...

Confirmation Received from MHLW That QRX003 Qualifies for Both Orphan Drug Designation and Fast Track Regulatory Review Status Quoin Initiating the Establishment of a Japanese Subsidiary to...