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 ...

Consensus directed medical therapy (CDMT) was used in 73.1% of DMD patients with moderate or severe left ventricular dysfunction, leaving nearly 30% without recommended treatment. Target doses were ...

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 ...

Duchenne and Becker muscular dystrophy are similar conditions caused by mutations in the same gene. However, Duchenne muscular dystrophy begins to cause symptoms at an earlier age, is more severe, and ...

Melanie Sanford was worried that her son, Hudson, wasn’t jumping around as much as other toddlers his age. But his pediatrician reassured her that kids all develop at their own pace, and “he wasn’t ...

—Surveys of caregivers and health care providers reveal gaps in screening and management of neuropsychiatric and neurodevelopmental symptoms among patients with Duchenne muscular dystrophy in the ...

Muscle weakness in Duchenne muscular dystrophy can lead to symptoms related to mobility, coordination, posture, and breathing, as well as other health effects. Duchenne muscular dystrophy (DMD) is a ...