Treatment advances for Duchenne muscular dystrophy
Alexander Zygmunt1,2, Cuixia Tian1,2
1Cincinnati Children's Hospital Medical Center.
Current Opinion in Pediatrics
|April 1, 2026
Summary
Recent advancements offer new hope for Duchenne muscular dystrophy (DMD) patients with seven novel medications approved since 2016. These treatments target different pathways to combat progressive muscle wasting in this severe genetic disorder.
Area of Science:
- Neurology
- Genetics
- Pharmacology
Background:
- Duchenne muscular dystrophy (DMD) is a severe X-linked muscle wasting disease.
- Historically, treatment options were limited to glucocorticoid steroids.
- Recent years have seen significant advancements in pharmacologic interventions for DMD.
Purpose of the Study:
- To review novel pharmacologic interventions for Duchenne muscular dystrophy.
- To summarize mechanisms of action, safety, and efficacy of new DMD medications.
- To provide an overview of recent FDA-approved treatments for DMD.
Main Methods:
- Literature review of recently approved DMD medications.
- Analysis of drug mechanisms targeting DMD disease pathways.
- Synthesis of safety data and motor function impact evidence.
Main Results:
- Seven new medications approved by the FDA since 2016.
- Includes novel glucocorticoids (vamorolone), exon-skipping therapies (eteplirsen, golodirsen, vitolarsen, casimersen), gene therapy (delandistrogene moxeparvovec), and HDAC inhibitors (givinostat).
- These therapies target various aspects of the DMD disease process.
Conclusions:
- Novel medications offer diverse therapeutic strategies for DMD.
- Understanding mechanisms, safety, and functional outcomes is crucial.
- These advancements represent a significant shift in DMD treatment paradigms.
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