Corticosteroids for Duchenne Muscular Dystrophy: Translating Evidence into Practice

Updated guidance from the American Academy of Neurology highlights the benefits of corticosteroids for boys with DMD.

Duchenne muscular dystrophy (DMD) is the most common childhood muscular dystrophy (annual incidence, about 1 in 5000 male live births). In the disorder's devastating natural history, boys lose the ability to ambulate at a mean age of 9 years and die at a mean age of 19 years. Although DMD patients have long been known to benefit from corticosteroids, widespread variation in practice persists — and as many as 20% of boys do not receive this treatment.

A recent update to the American Academy of Neurology practice guideline underscores the specific benefits of corticosteroids, highlights current gaps in our knowledge, and places these in the context of clinical care. Key recommendations are as follows:

Prednisone should be used to improve streng…

Reviewing Author

Michael Benatar, MD, MS, PhD

Disclosures

Grant / Research supportNIH NeuroBioBank; ALS Association; NIH/National Institute of Neurological Disorders and Stroke; NIH/National Center for Advancing Translational Sciences; FDA; Department of Defense

Editorial boardsCochrane Collaboration

Leadership positions in professional societiesMuscle Study Group Executive Committee

Michael Benatar, MD, MS, PhD

Disclosures

Editorial boardsCochrane Collaboration

Leadership positions in professional societiesMuscle Study Group Executive Committee

Corticosteroids for Duchenne Muscular Dystrophy: Translating Evidence into Practice

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Reviewing Author