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

At the 44th Annual J.P. Morgan Healthcare Conference in early January 2026, Edgewise Therapeutics outlined its 2026 clinical ...

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 ...

A Sarepta Therapeutics gene therapy that failed its confirmatory test has now converted its accelerated FDA approval into a traditional one, expanding use of the treatment to a wider group of Duchenne ...

Commercial launch in Switzerland anticipated in H2 2026Pratteln, Switzerland, January 15, 2026 – Santhera Pharmaceuticals (SIX: SANN) announces ...

Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...

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 ...

Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation ...

If cancer is a disease of overabundance, where cells divide without restraint and tumors grow despite the body's best interests, then degenerative diseases are disorders of deprivation. When ...

The Muscular Dystrophy Association (MDA) is proud to announce the recipients of the 2026 MDA Legacy Awards, recognizing excellence in scientific and clinical research and saluting outstanding ...

In research published in Developmental Medicine & Child Neurology, investigators have developed a brief, reliable, and valid ...

Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...