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 ...

DMD, a genetic disorder affecting 1 in 5000 male births, is caused by mutations in the DMD gene. While it primarily leads to ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort.

Get Instant Summarized Text (Gist) Gene therapy for Duchenne muscular dystrophy (DMD) faces challenges as the immune system may hinder its effectiveness. A large-scale trial showed that the ...

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

Exon skipping is one approach to treat DMD that involves using small, synthetic DNA molecules called antisense ... stop codon into a patient’s mRNA sequence, causing the production of truncated ...

Sarepta Therapeutics said it will update its prescribing information for Elevidys® (delandistrogene moxeparvovec-rokl) after acknowledging the sudden death of a patient with Duchenne muscular ...

Opens in a new tab or window A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta ...

One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech said (PDF) Tuesday. The patient suffered ...

DMD, a genetic disorder affecting 1 in 5000 male births, is caused by mutations in the DMD gene. While it primarily ... Using RNA sequencing and functional brain analyses the team of 21 scientists ...

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