Treatment with the investigational agent givinostat has the potential to delay disease progression when added to corticosteroid treatment in ambulatory boys with Duchenne muscular dystrophy.
compared with placebo, including reduced decline of muscle function and strength, results from the phase 3 EPIDYS trial show.
DMD is a "devastating disease that has only limited treatment options available," Trudeau noted. "If givinostat is approved by the FDA, it could provide a large number of patients with a daily, orally administered therapeutic that could represent a significant advance in treatment of DMD." Givinostat inhibits histone deacetylases , enzymes that modulate gene and protein expression in muscle.
A total of 118 patients received oral givinostat, and 61 received matching placebo twice daily along with corticosteroid treatment for 72 weeks. They were evaluated every 12 weeks for 72 weeks.Treatment with givinostat was associated with a slower decline in performing the four-stair climb compared with the placebo group , a 17-item rating scale that measures motor function, compared with placebo.
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