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 ...

An experimental gene therapy for Duchenne Muscular Dystrophy (DMD) has showed better-than-expected results in a three-patient trial, according to preliminary data. Company shares jumped 60 percent ...

has established a gene therapy centre to treat rare genetic diseases such as Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD). The announcement was made at the ongoing Arab ...

Two years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl), motor function continues to improve in boys with Duchenne muscular dystrophy (DMD) who entered a global ...

INS1201 represents the first application of Insmed’s targeted AAV delivery platform in DMD. Credit: JHVEPhoto / Shutterstock. Duchenne muscular dystrophy (DMD) is a rare genetic disorder that is ...

NEW YORK – Sarepta Therapeutics on Monday presented top-line results from the second part of its global, randomized-controlled Phase III EMBARK trial, which company executives said reinforce earlier ...

Duchenne muscular dystrophy (DMD) is the most common type. It’s caused by flaws in the gene that controls how the body keeps muscles healthy. The disease almost always affects boys, and symptoms ...

Sidra Medicine, a member of Qatar Foundation, has established a Gene Therapy Center to treat rare genetic diseases such as Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD). The ...

An inherited disorder affecting primarily boys, DMD is characterized by the progressive loss of skeletal, heart and lung muscle. It is caused by a mutation in the gene for the dystrophin protein, ...

For patients with Duchenne muscular dystrophy, the last decade has been a fruitful landscape of progress. Eight drugs have been approved in the last eight years—including Sarepta’s Elevidys as the ...

(IN BRIEF) Roche has reported positive results from the second year of the EMBARK Phase III trial of Elevidys (delandistrogene moxeparvovec), the first approved gene therapy for Duchenne muscular ...