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

EP: 1.Clinical Presentation and Diagnosis of Duchenne Muscular Dystrophy

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

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

EP: 4.Gene Therapy for Treatment of Duchenne Muscular Dystrophy

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

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

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?