Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the ...

The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...

Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key functions of full-length dystrophin and allows it be delivered more effectively ...

Sarepta Therapeutics ... It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 ...

Utrophin increase in muscle cells normalises cell function in Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is chronic and begins in childhood. The life expectancy of affected patients ...

Older age, presence of scoliosis, and other factors were associated with lower predicted forced vital capacity in patients with Duchenne muscular dystrophy using non-invasive ventilation.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report fourth ...

The company has several drugs on the market that treat Duchenne muscular dystrophy, a progressive genetic disease that weakens patients' muscles. The most important therapy in Sarepta Therapeutics ...