Sarepta Therapeutics has come under pressure following the deaths of three patients using its treatments.

Sarepta stock popped Wednesday after the company reported promising results for its gene-silencing tech in two forms of muscular dystrophy.

"High clinical suspicion is important for this population of patients. Since muscular dystrophy is progressive in nature, monitoring disease progression in these patients is critical for timing ...

Becker muscular dystrophy (BMD) is a type of muscular dystrophy, a genetic condition that weakens and damages muscles. It can worsen with age. However, its symptoms are less severe than those of ...

Recent studies have defined a group of muscular dystrophies, now termed the dystroglycanopathies, as novel disorders of glycosylation. These conditions include Walker–Warburg syndrome, ...

Sarepta Therapeutics (NASDAQ:SRPT) stock is up approximately 20% in early trading on Wednesday after the company released its ...

Using brain organoids, researchers discover mutational commonalities between muscular dystrophy type 1 and Rett syndrome, suggesting the potential of a similar treatment for both. Myotonic dystrophy ...

CORAL GABLES, Fla. — There’s new hope for children born with a fatal form of muscular dystrophy. Nicklaus Children’s Hospital is one of the first in the nation and one of only three hospitals in ...

PALO ALTO, Calif., June 26, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced ...