"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 ...

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

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

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.

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 ...

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 ...

Duchenne and Becker muscular dystrophy are similar conditions caused by mutations in the same gene. However, Duchenne muscular dystrophy begins to cause symptoms at an earlier age, is more severe, and ...

A study by Indiana University School of Medicine researchers sheds new light on the development and treatment of a rare form of muscular dystrophy. The study's findings were recently published in ...

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 ...