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Daily steroid use brings longer ambulation time but also more adverse effects, except for orthopaedic complications
Duchenne muscular dystrophy (DMD) is an X-linked muscular dystrophy known to affect one in 3600 live male births and leads to progressive proximal muscle weakness in the first years, and orthopaedic, respiratory and cardiac complications in the teens. Typically the patients would lose the ability of ambulation at around the age of 10 years and decease in young adulthood if untreated. In recent years, corticosteroid treatment has been proved to prolong ambulation, improve quality of life and survival. Moreover, it has also shown efficacy in reducing the degree of scoliosis and improving cardiopulmonary function. …
Competing interests None.
Provenance and peer review Commissioned; externally peer reviewed.