Dystrophinopathies Medication

Updated: Nov 13, 2019
  • Author: Dinesh G Nair, MD, PhD; Chief Editor: Nicholas Lorenzo, MD, CPE, MHCM, FAAPL  more...
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Medication Summary

To date, prednisone is the only medication that has demonstrated even a modest benefit in modifying the course of the disease. Clinical improvement is seen as early as 1 month after starting treatment and lasts as long as 3 years. Children who discontinue steroids for various reasons soon revert to natural downward progression of the disease. Please refer to the supportive therapies section.



Class Summary

These agents have anti-inflammatory properties and cause profound and varied metabolic effects. Corticosteroids modify the body's immune response to diverse stimuli.

Prednisone (Deltasone, Orasone, Meticorten, Sterapred)

Anti-inflammatory drug with unknown mechanism of action.