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Medpage Today on MSN18h
Bone Health in Duchenne Muscular Dystrophy
An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...
REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
Amazon S3 on MSN2d
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 ...
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 ...
Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...
Lancashire Evening Post on MSN3d
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 ...
Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s ...
Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...
European Medicines Agency recognizes potential of lead compound MP1032 in addressing a high unmet medical need in Duchenne Muscular Dystrophy (DMD)Designation is paving the way for safer long-term ...
The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
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