Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.
The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...
These include gene-based therapies (e.g., replacing a patient's faulty DMD genes with normally functioning ones), cell-based therapies (e.g., replacing dystrophin-deficient muscle cells with stem ...
Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...
Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...
One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech ...
New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety. Updated ...
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