Although a profound transformation has occurred in both survival and clinical course, long-term data on the efficacy and safety of disease-modifying therapies is required to inform their use in ...

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 ...

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that starts in childhood and causes muscle weakness that worsens over time. There’s no cure for DMD, but new treatments have helped slow ...

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Genetic testing can confirm a muscular dystrophy (MD) diagnosis when symptoms and other tests already suggest MD. It also identifies specific gene mutations that can guide targeted treatment. Genetic ...

Six-year-old José Capablanca was excited to see his visitors. “I think you guys want to know how fast I can run!” he shouted, racing around his family’s home in Gallatin, Tennessee. José has Duchenne ...

Becker muscular dystrophy (BMD) is a genetic disorder that causes progressive muscle degeneration and weakness. This can cause a person to have reduced mobility such as problems walking. BMD can make ...