FDA approves nonsteroidal treatment for Duchenne muscular dystrophy

The U.S. Food and Drug Administration has approved the oral drug Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy in patients 6 and older. It is the first nonsteroidal drug approved to treat patients with all genetic variants of the disease.

Duchenne muscular dystrophy is the most common childhood form of muscular dystrophy and typically affects males. It is a rare neurological disorder that causes progressive muscle weakness because of a lack of a muscle protein called dystrophin.

Duvyzat is a histone deacetylase inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle.

Read more: FDA

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