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.
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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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