For decades, there has been no cure and few treatment options.

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is linked ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer ...

A multidisciplinary team, usually led by a neurologist, treats muscular dystrophy (MD). They’ll tailor the type of treatment you receive to your specific needs. MD is a group of conditions that lead ...

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 ...

A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...

BUFFALO, N.Y. (WKBW) — Twin brothers Daniel and Matt Murray of Buffalo are preparing to take on three marathons together to raise awareness and funds for Duchenne muscular dystrophy, a condition Matt ...

In an eagerly anticipated decision, the Food and Drug Administration Thursday approved the first gene therapy for muscular dystrophy. "Today's approval addresses an urgent unmet medical need and is an ...

More than 30 rare muscular dystrophy types cause progressive muscle weakness from inherited gene mutations. Combined, they affect about 1 in 5,000 to 8,000 people. Common subtypes vary by age of onset ...

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 ...