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 ...
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 US HHS has added Duchenne muscular dystrophy and metachromatic leukodystrophy to newborn screening, citing benefits of early detection and access to FDA-approved treatments.
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 ...
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 ...
This is an archived article and the information in the article may be outdated. Please look at the time stamp on the story to see when it was last updated. There’s news of a potential treatment that ...
The Utah Program for Inherited Neuromuscular Disorders (UPIN) improves the lives of men, women, and children with inherited nerve or muscle disorders through cutting-edge, multidisciplinary care and ...
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 ...
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