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.

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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