The Phase I/II Deliver trial sought to establish the impact of DYNE-251 on several mobility-related endpoints, seeing an ...

RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, per interim data.

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Precision BioSciences (DTIL) announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of ...

A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with ...

yet functional dystrophin protein. The U.S. Food and Drug Administration (FDA) has granted PGN-EDO51 both Orphan Drug and Rare Pediatric Disease Designations for the treatment of patients with DMD ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

The company behind the drug, Sarepta Therapeutics, said that the patient suffered acute liver injury, a known side effect, ...

Sarepta shares fell after reporting a patient death linked to Elevidys. The case involved acute liver failure, with a CMV infection as a potential factor.

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