In an eagerly anticipated decision, the Food and Drug Administration Thursday approved the first gene therapy for muscular dystrophy. "Today's approval addresses an urgent unmet medical need and is an ...
Consensus directed medical therapy (CDMT) was used in 73.1% of DMD patients with moderate or severe left ventricular dysfunction, leaving nearly 30% without recommended treatment. Target doses were ...
Orphan Drug Designation (ODD) recognizes Duchenne muscular dystrophy as a rare disease with significant unmet medical need without an established ...
WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the ...
WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%. The young ...
The companies anticipate finalising the acquisition in the third quarter of 2026.
Edgewise Therapeutics (EWTX) stays a Buy after its $2.65B Servier deal funds EDG-7500 for HCM with key Q2 2026 data. Read ...
The ‘Protect Iliauni’ movement says GEL 2 million has been raised to support children with Duchenne muscular dystrophy. In a ...
Children's National Medical Center in Washington, D.C. is doing pioneering research and treatment for muscular dystrophy using interdisciplinary teams of doctors and scientists focused on a common ...
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