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Rare Disease

FDA approves DMD treatment from Italfarmaco

by Aayushi Pratap
March 28, 2024 | A version of this story appeared in Volume 102, Issue 10
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Most Popular in Pharmaceuticals

 

The structure of givinostat.

The US Food and Drug Administration has approved Duvyzat (givinostat) to treat Duchenne muscular dystrophy (DMD), a rare neuromuscular genetic condition. The condition leads to alterations in a protein called dystrophin that keeps muscle cells intact. Duvyzat, a small-molecule nonsteroidal drug, works by inhibiting histone deacetylase enzymes, thereby reducing inflammation and muscle loss. The drug, from the Italian firm Italfarmaco, is the third new treatment approved for the condition since June.

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