Duchenne muscular dystrophy

An X-linked recessive muscle disease caused by mutations in the dystrophin gene (Xp21). Onset at 2–5 years, progressive weakness, loss of ambulation usually by 10–12 years, with cardiomyopathy and respiratory failure later. Foundation therapy is glucocorticoids (deflazacort or prednisolone) from age 4–5: they slow loss of muscle function and reduce scoliosis (DMD Care Considerations 2018, Cochrane 2016). Compared with prednisolone, deflazacort causes less weight gain and fewer cushingoid features.

ICD-10 G71.01 drug

Also known as: DMD

Drugs used for this condition

ADeflazacortFirst line · Systemic glucocorticoid (oxazoline derivative)Drug of choice for Duchenne muscular dystrophy in boys from age 4–5. Per DMD Care Considerations 2018 and AAN 2023, deflazacort slows loss of muscle function, delays loss of ambulation by 2–3 years, and reduces scoliosis risk. Versus prednisolone it produces fewer cushingoid features and less weight gain, so Spain and Italy often prefer it. Dose 0.9 mg/kg/day, lifelong, with scheduled monitoring of growth, bone density, and vision.

How to read tiers A–F

Evidence tiers belong to the drug-indication pair, not to the drug as a whole: the same substance can be A for one condition and F for another.

AStrong evidence – first or second line in international guidelines
BRecommended in selected cases
CStudy data contradict each other
DBenefit not proven, no documented harm
FDocumented harm, or risk without benefit