The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...

Scientists have been searching for ways to cure DMD for as long as they have known about the disease. Today, based on the wealth of knowledge about the underlying genetic and molecular mechanisms ...

Ottawa Senators defenseman Nick Jensen's wife Jenner has opened up about the couple's personal fight against Duchenne muscular dystrophy, a genetic disorder that causes progressive muscle ...

Duchenne Muscular Dystrophy (DMD) is a rare progressive disorder. People with DMD are missing muscle-protecting protein. This is due to a fault in the gene producing dystrophin. Muscles to become ...

As the Connecticut legislature meets for the 2025 session, it has the task to add one more disease, Duchenne muscular dystrophy, to the state’s newborn screening panel. A bipartisan group of six ...

Sarepta's exon-skipping therapies for DMD – Exondys 51 ... 2a proof-of-concept data for its therapy for irritable bowel syndrome (IBS) based on gut microbiome species.

DMD is a rare, inherited condition characterized ... atypical hemolytic uremic syndrome, or hemolysis. Notably, none of the AEs seen required the use of immunomodulatory agents such as eculizumab ...