Some muscular dystrophies are mild. Others are more serious and can cause life-threatening muscle weakness. Most children with Duchenne muscular dystrophy ... weakness in the shoulders, upper ...

Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to ...

Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...

Duchenne Muscular Dystrophy (DMD) is a rare progressive disorder. People with DMD are missing muscle-protecting protein. This is due to a fault in the gene producing dystrophin. Muscles to become ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). DMD, a severe genetic disorder that causes muscle weakness and shortens lifespans, arises from mutations in ...

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...

Sarepta Therapeutics has decided not to continue the development of vesleteplirsen, a follow-up to its Duchenne muscular dystrophy (DMD) therapy Exondys 51. The drug – formerly known as SRP-5051 ...

Solid Biosciences ended Q3 2024 with $171.1 million in cash, securing operations through 2026. Initial Phase 1/2 trial results for SGT-003 in DMD expected in Q1 2025. Wedbush initiated coverage on ...