Sarepta Therapeutics has provided additional safety data on its gene therapy Elevidys as a Duchenne muscular dystrophy ...

The deaths prompted the FDA to ask Sarepta to stop shipping Elevidys for all patients, but the company refused to do so. Days later, it changed tack in order to maintain what it said was a “positive ...

Jefferies analysts said these detailed safety outcomes confirm the gene therapy's positive risk/benefit profile in ambulatory ...

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

FDA Informs Sarepta That It Recommends That Sarepta Remove Its Pause and Resume Shipments of ELEVIDYS for Ambulatory Individuals With Duchenne Muscular Dystrophy ...

ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene ...

Sarepta Therapeutics said Friday that the deaths of two patients taking its marketed gene therapy Elevidys® (delandistrogene ...

Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

The biotech, which cut jobs following the rejection in May of its Barth syndrome therapy, claimed it has addressed the agency ...

ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age.

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.