"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 ...

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 ...

The FDA has put a partial clinical hold on PepGen’s phase 2 mus | The FDA has put a partial clinical hold on PepGen’s phase 2 muscle wasting trial after reviewing preclinical data, continuing the stop ...

A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...

Becker muscular dystrophy (BMD) is a genetic disorder that causes progressive muscle degeneration and weakness. This can cause a person to have reduced mobility such as problems walking. BMD can make ...

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 ...

Del-desiran reduced DMPK mRNA levels, but led to two serious adverse events ...

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

On March 10, 2026, from 12:00 p.m. -- 1:30 p.m. ET, in room Key West ABCD at the Hilton Orlando, Avidity will host an industry forum lunch titled "Biomarker Advancements in Rare Neuromuscular Disease: ...