After Sarepta reported the death of a patient who had recently taken the gene therapy Elevydis, patient advocacy group Parent ...
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New Treatment Strategy for Rare Disease Dutsenic Muscle Dystrophy...EZH2 inhibitor steroid effective in combinationKorean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...
The firm's stock price dropped 27 percent on Tuesday following the news that the young man died from acute liver failure after getting Elevidys.
Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...
Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...
REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...
A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...
Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease----Data Presented at the 2025 Muscular ...
Solid Biosciences stock soars 60% in a month after the company announced positive initial data from its phase I/II DMD study ...
About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular ... function and repair of muscle cells. In DMD, muscle fibres are highly susceptible to injury and this continuous muscle ...
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