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 ...
The agency acted after reviewing mouse data submitted in 2024, making the timing of the new pause “confusing,” one analyst wrote.
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 ...
A RECOMMENDATION for the HSE not to cover the cost of a drug to help children with a rare muscular disease has been blasted by families. Last year, The Irish Sun highlighted the plight of ...
For sura-vec in wet AMD, top line data from pivotal studies ATMOSPHERE and ASCENT are anticipated in Q4. First patient dosing in NAVIGATE for diabetic retinopathy is planned next quarter, expected to ...
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 ...
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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