Data from the EMBARK trial suggest patients may have functional improvements taking the gene therapy between 8 and 9 years old.

Sarepta Therapeutics is meeting with the FDA in June to discuss expanding the use of its Duchenne muscular dystrophy gene ...

Since Elevidys' accelerated approval in 2023, experts have been clamoring for more data, particularly in older and ...

Rocket Pharmaceuticals has shared early data from three patients that tie its gene therapy for an inherited heart disease to ...

Groundbreaking gene therapy offers hope for a boy with fatal muscular dystrophy, potentially slowing disease progression and ...

GlobalData analysts have tipped the global market for genomic therapies to balloon to almost $90 billion by 2030, driven by ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular ...

Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients ...

The approval was based on the therapy’s safety and efficacy data, including muscle health and longer-term functional outcomes ...

It has developed several medicines for Duchenne muscular dystrophy (DMD), a progressive, genetic, muscle-wasting disease. One of the company's most important products is Elevidys, a gene therapy for ...