DMD – corticosteroids — RACP Paediatrics MCQ

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ModerateNeurologyDMD – corticosteroidsRACP Paediatrics

A 6-year-old with confirmed Duchenne muscular dystrophy (DMD) is ambulant with Gowers sign and progressive proximal weakness. What is the most important pharmacological treatment to slow disease progression?

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Correct answer: D — Oral prednisolone/deflazacort

Corticosteroid therapy (prednisolone or deflazacort) is the standard of care for DMD. It prolongs ambulation by approximately 2-3 years, preserves respiratory and cardiac function, and reduces scoliosis risk. Treatment is typically started when motor skills plateau (usually 4-7 years). Side effects (weight gain, short stature, behavioural changes, osteoporosis) require monitoring.

Reference: RCH Melbourne – 2023 – DMD CPG; RACP Paediatric Curriculum – Neurology