Abstract
Givinostat (Duvyzat ā Italfarmaco), an oral histone deacetylase inhibitor, has been approved by the FDA for treatment of patients ā„6 years old with Duchenne muscular dystrophy (DMD), regardless of the DMD-causing mutation. It is the first nonsteroidal drug to be approved for this indication. The oral corticosteroids deflazacort (Emflaza) and vamorolone (Agamree) are also approved for treatment of patients with all genetic variants of DMD.
Published Version
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