Genethon is determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic ...

European Medicines Agency recognizes potential of lead compound MP1032 in addressing a high unmet medical need in Duchenne Muscular Dystrophy (DMD)Designation is paving the way for safer long-term ...

Dr. Vinay Prasad, who came under fire from patient advocacy groups and Laura Loomer, is gone after less than three months on ...

Sarepta Therapeutics’ stock was soaring Tuesday after the FDA recommended lifting the pause on the company’s Duchenne ...

Quantitative muscle ultrasound correlates strongly with ambulatory and timed function tests in Duchenne muscular dystrophy, ...

Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...

Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...

Sarepta stock sees upgrades and price hikes as analysts reassess Elevidys prospects following a favorable FDA development.

Stock of Sarepta Therapeutics Inc. (NASDAQ: SRPT) surged 38.89% in Tuesday pre-market following the U.S. Food and Drug ...