"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 ...

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 ...

Zeleciment basivarsen (z-basivarsen) demonstrated sustained functional improvement across multiple clinical measures in the ongoing ACHIEVE ...

-- Continued functional improvements wereobserved at 18 months in the low-dose cohort-- -- First look at functional outcomes in high-dose cohort found improvements 6 months after administration -- -- ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

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 ...

EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting muscular dystrophies and cardiomyopathies, today ...

Duchenne Muscular Dystrophy (DMD) is a debilitating genetic disorder that eventually leaves patients in a wheelchair. In a new study, researchers have found that an existing cancer drug shows promise ...

A person may inherit the genetic changes responsible for muscular dystrophy. These genetic changes can also occur due to spontaneous genetic mutations. In either case, the disease is not preventable.

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 ...

Oculopharyngeal muscular dystrophy (OPMD) is a rare inherited disorder that causes weakness in the eye and throat muscles. It may lead to drooping eyelids and swallowing difficulties. Muscular ...

EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and ...