Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...
The company is evaluating whether the drug, which targets cardiac complications, might be used treat patients with other ...
There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results ...
Others are more serious and can cause life-threatening muscle weakness. Most children with Duchenne muscular dystrophy will need a wheelchair to get around by the time they're teenagers.
Limb-girdle muscular dystrophy type 2D (LGMD2D/R3) is a rare genetic disorder caused by mutations in the SGCA gene, leading to defective folding and the loss of functional α-sarcoglycan, with ...
chief research officer of the Muscular Dystrophy Association in Chicago. Here's what myasthenia gravis is, what causes it, and what treatment options exist if you or someone you love is affected.