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

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

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 ...

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

After our editorial, the agency relents to allow a Duchenne treatment.

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

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

Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...

The U.S. Food and Drug Administration (FDA) announced on Friday, after market close, that it is investigating the death of an ...

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.

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