A young man with Duchenne muscular dystrophy (DMD) who received the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) has died due to acute liver failure. In a statement, the ...
(AP Photo/Manuel Balce Ceneta, File) Updated [hour]:[minute] [AMPM] [timezone], [monthFull] [day], [year] WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its ...
A patient has died following treatment with Elevidys, the Sarepta Therapeutics product that is the only FDA-approved gene therapy for Duchenne muscular dystrophy, the company disclosed Tuesday.
its FDA-approved treatment for Duchenne muscular dystrophy. In a statement, the Cambridge, Massachusetts-based biotech said that a young man with DMD died after receiving Elevidys, a gene therapy ...
The mum of an 11-year-old boy who is waiting to find out if he can access a new drug to treat his Duchenne muscular dystrophy (DMD) said her son talked about his fear of death. Harry, from Corby ...
A close friend of 30 years recently asked what my life was like before I was diagnosed with limb-girdle muscular dystrophy. We then talked about what I’d already known when I was diagnosed in 1985, as ...
About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular disorder caused by a mutation in the DMD gene which affects the production of a protein called dystrophin. 3 Dystrophin is a ...
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