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Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy

Muscle & Nerve Feb 27, 2019

Connolly AM, et al. - In boys with Duchenne muscular dystrophy (DMD), glucocorticosteroids (GC) effectively slows weakness, so researchers tested the safety and efficacy of twice-weekly prednisolone (5 mg/kg/dose) in infants and young boys (0.4–2.4 years) with DMD. On the Bayley-III gross motor scaled score (GMSS), the treated patients gained an average of 0.5 points vs the historical control cohort, which averaged a 1.3 point decline. Investigators found that twice-weekly GC was well tolerated in infants and young boys with DMD and improves GMSS. A possible risk was excessive weight gain.

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