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An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...
Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.
REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...
Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...
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The story of Sarepta's Duchenne gene therapy
The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
FDA permits use of Sarepta Therapeutics’ Duchenne therapy in younger patients after short-lived halt
The FDA said it was clearing the way for Sarepta Therapeutics to resume shipments of its gene therapy for Duchenne muscular ...
Lancashire Evening Post on MSN2d
Cumbria family’s ‘Iron Will’ triathlon for son living with muscular dystrophy
On Thursday 28 August, Sam Taylor, 46, his wife Sue, 47, and daughter Beth, 17, from Crooklands in Cumbria will set off to ...
The U.S. Food and Drug Administration (FDA) announced on Friday, after market close, that it is investigating the death of an ...
Quantitative muscle ultrasound correlates strongly with ambulatory and timed function tests in Duchenne muscular dystrophy, ...
Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...
Sarepta Therapeutics’ stock was soaring Tuesday after the FDA recommended lifting the pause on the company’s Duchenne ...
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