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Opinion|Videos|July 10, 2024

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

Author(s)Emma Ciafaloni, MD

Emma Ciafaloni, MD, discusses therapies for Duchenne muscular dystrophy that target pathways downstream of dystrophin, focusing on vamorolone and givinostat.

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:

Discuss the efficacy and safety of vamorolone, a corticosteroid, which was FDA approved in 2023 for patients with Duchenne muscular dystrophy (DMD) aged 2 years and older.
Where does givinostat, a pan-histone deacetylase inhibitor, that was FDA approved in 2024, fit in the treatment landscape of DMD?

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