Opinion|Videos|July 10, 2024

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

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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