Duchenne muscular dystrophy medical therapy

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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

Duchenne muscular dystrophy

  • 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

  1. Finkel RS (September 2010). "Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124)". J. Child Neurol. 25 (9): 1158–64. doi:10.1177/0883073810371129. PMC 3674569. PMID 20519671.
  2. Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, Dickson G, Wood MJ, Wilton SD, Straub V, Kole R, Shrewsbury SB, Sewry C, Morgan JE, Bushby K, Muntoni F (August 2011). "Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study". Lancet. 378 (9791): 595–605. doi:10.1016/S0140-6736(11)60756-3. PMC 3156980. PMID 21784508.

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