Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today presented at the 2026 MDA Clinical and Scientific Conference, long-term data from its MESA ...

ITF Therapeutics LLC, the U.S. rare disease affiliate of Italfarmaco, today announced the presentation of ten abstracts at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference ...

Interim observations from the Phase 2 TRAILHEAD study show continued improvement in handgrip strength, overall stability of elbow and shoulder ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

Seven-year-old William, a Southern Oregon boy living with Duchenne muscular dystrophy, is going to get a never-before-tried surgery designed specifically for his genetic mutation. NBC5’s medical ...

HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 -- 48-week trial will enroll approximately 150 ...

A RECOMMENDATION for the HSE not to cover the cost of a drug to help children with a rare muscular disease has been blasted by families. Last year, The Irish Sun highlighted the plight of ...

Muscular dystrophy (MD) is a group of genetic diseases that cause your muscles to progressively weaken and degenerate. There are several types of MD, each with its own symptoms, but they all involve ...

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 muscular dystrophy has several approved drugs, including a gene therapy that provides children who have the rare, inherited muscle-wasting disease the option of a one-time treatment. But each ...

William is a seven-year-old boy from Southern Oregon living with Duchenne muscular dystrophy. Now, he is preparing to undergo a surgery that has never been tried before, a procedure designed ...