Opinion
Video
Understanding the Causes of Duchenne Muscular Dystrophy
Author(s):
A panelist discusses how steroid use in DMD patients provides significant benefits in delaying disease progression, particularly loss of ambulation, despite potential side effects including mood disorders, bone weakness, and weight gain.
Clinical Brief: Steroid Therapy Benefits and Limitations in DMD
Main Discussion Topics
- Prednisone and deflazacort demonstrate significant delay in disease progression
- Potential side effects including mood disorders, bone weakness, weight gain, and stunted growth
- Individualized risk-benefit assessment for continued steroid therapy
- Long-term impact on respiratory and cardiac function less clearly established
Key Points for Physicians
- Regular steroid use can add several years of maintained mobility and independence
- Monitor for behavioral changes and hyperactivity which may limit tolerability
- Risk of fractures increases with long-term use, particularly in older boys
- Dosing regimens (daily vs. weekly) should be individualized based on response and side effects
Notable Insights
While steroid therapy remains a cornerstone of treatment, the balance between functional benefits and side effects requires ongoing assessment. Tolerability varies significantly between patients, necessitating personalized approaches.
Clinical Significance
Despite potential adverse effects, corticosteroid therapy provides substantial benefit in delaying disease progression, with the greatest evidence for preserved ambulation but likely additional benefits for respiratory and cardiac function that require further study.
Newsletter
Keep your finger on the pulse of neurology—subscribe to NeurologyLive for expert interviews, new data, and breakthrough treatment updates.
