An experimental RNA therapy from Avidity Biosciences has early clinical trial results showing it reduced by half the expression of a gene at the root of a rare, inherited form of muscular dystrophy ...

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 ...

One of the first symptoms of LGMD is typically a weakening of the muscles in the hip and shoulder girdles. The hip girdle is a ring-like bone structure in the pelvic area, consisting of the pelvis, ...

A regularly prescribed class of heart medications might be capable of treating one of the most common forms of muscular dystrophy, a new study in mice suggests. Myotonic dystrophy type 1 (DM1) is ...

Muscular dystrophy, known as DMD, is a rare and fatal genetic disease that primarily affects boys, slowly robbing them of their ability to walk, breathe and live independently. Until now, there have ...

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

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...