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Updated: Feb 8, 2026
09:18
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
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Current and Emerging Therapies for Duchenne Muscular Dystrophy
Megan Crone1,2, Jean K Mah3
1Division of Neurology, Department of Pediatrics, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada. hollanmt@ucalgary.ca.
Current Treatment Options in Neurology
|June 25, 2018
Summary
Emerging therapies for Duchenne muscular dystrophy (DMD) show promise in treating the underlying disease and its symptoms. These advancements, alongside supportive care, aim to improve the quality of life for individuals with DMD.
Area of Science:
- Neurology
- Genetics
- Pharmacology
Background:
- Duchenne muscular dystrophy (DMD) is a severe, progressive genetic disorder affecting muscle function.
- Standardized care guidelines are evolving alongside new therapeutic strategies for DMD.
Purpose of the Study:
- To review current and emerging therapies for Duchenne muscular dystrophy (DMD).
Main Methods:
- Literature review of preclinical and clinical studies on DMD therapies.
- Analysis of emerging treatment modalities targeting pathobiology and symptoms.
Main Results:
- Several therapeutic approaches, including gene replacement and exon skipping, show variable success.
- Symptomatic treatments for ischemia, regeneration, fibrosis, myostatin inhibition, and inflammation are under investigation.
- DMD is a complex disease with diverse therapeutic targets.
Conclusions:
- Recent pharmacological and technological advancements offer improved quality of life for DMD patients.
- Timely supportive interventions are crucial for managing Duchenne muscular dystrophy.
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