Glucocorticoid Corticosteroids for Duchenne Muscular Dystrophy

    Adnan Y. Manzur, Thierry Küntzer, Mike Pike, A V Swan
    TLDR Glucocorticoid corticosteroids improve muscle strength in Duchenne muscular dystrophy but can cause side effects.
    The review assessed the effects of glucocorticoid corticosteroids on boys with Duchenne muscular dystrophy (DMD), focusing on muscle strength and walking ability. It included six randomized controlled trials with a total of 249 participants. The findings indicated that glucocorticoid corticosteroids improved muscle strength and function over six months, with one trial showing stabilization for up to two years. The most effective regimen was prednisolone at 0.75 mg/kg/day. However, adverse effects such as weight gain, behavioral changes, and cushingoid appearance were more common compared to placebo. Long-term effects could not be evaluated due to the short duration of the studies, but non-randomized studies suggested both functional benefits and significant adverse effects with long-term use.
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