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Opinion|Videos|July 3, 2024

Current and Emerging Treatment Options and Management of Duchenne Muscular Dystrophy

Author(s)Emma Ciafaloni, MD

An expert on Duchenne muscular dystrophy reviews standard-of-care treatment options and provides clinical insights on adverse effects and safety concerns, particularly with corticosteroids.

Episodes in this series

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 1 Clinical Presentation and Diagnosis of Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 2 Communication of Duchenne Muscular Dystrophy Diagnosis and Coordination of Care

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 3 Current and Emerging Treatment Options and Management of Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 4 Gene Therapy for Treatment of Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 5 Targeting Pathways Downstream of Dystrophin: Vamorolone and Givinostat in Duchenne Muscular Dystrophy

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 6 Improvement in Management of Duchenne Muscular Dystrophy in Recent Years

Emma Ciafaloni, MD, an expert on Duchenne muscular dystrophy

EP. 7 Multidisciplinary Care in Duchenne Muscular Dystrophy

Video content above is prompted by the following questions:

Please briefly describe the current standard of care for Duchenne muscular dystrophy (DMD).
- What are the side effects and/or safety concerns associated with long-term use of corticosteroids in patients with DMD?
- Discuss recent approvals and ongoing studies in DMD.

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