This story is free to read because readers choose to support LAist. If you find value in independent local reporting, make a donation to power our newsroom today. In an eagerly anticipated decision, ...
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 ...
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 ...
Working with Parent Project Muscular Dystrophy and the Duchenne Registry on patient identification and trial awareness efforts ~15,000 children ...
Andelyn Biosciences, a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced today a partnership with Evolyra Therapeutics to ...
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 ...
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) — 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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