The EMA has called a temporary halt to a phase 2 trial of Sarepta's Roche-partnered Duchenne muscular dystrophy (DMD) gene therapy Elevidys in the wake of the death of a patient who received the ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...

Opens in a new tab or window A young person with Duchenne muscular dystrophy died following treatment with the recently ... in the DMD gene that leads to a lack of dystrophin and muscle loss.

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.

Wave Life Sciences’ Phase II open-label trial investigating its disease-modifying drug for boys living with Duchenne Muscular ...

One patient with Duchenne muscular dystrophy (DMD ... increase reflects the high unmet need in the disease and the lack of treatment options. Sarepta’s commercial partner, Roche, recorded ...

New Delhi: Over 200 parents from 26 states across India protested at Jantar Mantar on Wednesday with their children affected by Duchenne Muscular Dystrophy (DMD) and Muscular Dystrophy (MD ...

Results from the Forward-53 trial (NCT04906460) show the exon-skipping oligonucleotide-based therapy improved ... Typically, DMD develops due to a lack of the protein dystrophin, which helps ...