Duchenne muscular dystrophy medical therapy

Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Fahimeh Shojaei, M.D.

Overview

Pharmacologic medical therapies for Duchenne muscular dystrophy include glucocorticoids and disease-modifying treatment.

Medical Therapy

Pharmacologic medical therapies for Duchenne muscular dystrophy include glucocorticoids and disease-modifying treatment.^[1]^[2]

1 Glucocorticoids
- Preferred regimen : Prednisone 0.75 mg/kg PO per day q24h

2 Disease-modifying treatment
- Preferred regimen (1): Eteplirsen 30 mg/kg IV per week (infusion over 35 to 60 minutes)
- Preferred regimen (2): Ataluren 40 mg/kg PO per day q8h (10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening)

References

Overview