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Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...
The FDA greenlit multiple new drugs this month and issued some notable label expansions including for Eli Lilly’s Kisunla.
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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 ...
MyHealthTeam, a Swoop company, the creator of one of the largest, engaged patient social networks in healthcare, has announced the launch of myMDteam. This new website and patient community is ...
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
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 ...
A senior official at the US Food and Drug Administration has suddenly resigned after only three months in the job. Vinay Prasad’s departure followed controversy over a treatment for Duchenne muscular ...
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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 ...
Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...
Quantitative muscle ultrasound correlates strongly with ambulatory and timed function tests in Duchenne muscular dystrophy, ...
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