"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 ...

Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, ...

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 ...

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 ...

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 ...

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

There is no cure for DMD, but treatments can slow the progression of the disease and help improve quality of life. Duchenne muscular dystrophy (DMD) is a chronic condition that causes a gradual loss ...

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 ...

"Muscular dystrophy can take many things from me, but it will not take away my happiness, nor will it take away my faith." ...

FDA and Capricor aligned on endpoints for HOPE-3 pivotal trial HOPE-3 pivotal trial completed; topline data expected mid-Q4 2025 to support BLA resubmission Company preparing to resubmit CRL response ...