Phase 3 RCT: Ataluren shows favorable trend for slowing functional decline in nonsense mutation DMD

WHAT IS THIS SUMMARY ABOUT?: This article describes results from Study 041. Study 041 was a clinical study of ataluren, a treatment for people living with nonsense mutation Duchenne muscular dystrophy (nmDMD for short). Over time, people living with nmDMD experience muscle loss and reduced muscle strength and function (decline in muscle function). The researchers wanted to know whether 72 weeks of treatment with ataluren slowed the decline in muscle function in a large group of people living with nmDMD. To determine this, they compared the effects of ataluren with the effects of a placebo, which is a treatment that looks the same as the study treatment but has no active ingredients. The study also looked at the safety of ataluren over the 72 weeks of treatment. Ataluren has previously been compared with placebo in 48-week long clinical trials. WHAT WERE THE RESULTS?: In this study, 359 people took at least one dose of ataluren or placebo, even if they later switched or didn't stick to the treatment plan. Over 72 weeks, ability to walk and physical function declined less in people receiving ataluren than in people receiving placebo. This means that their physical abilities were maintained for longer if they took ataluren. The number of side effects were generally similar between people who took ataluren and those who took placebo. WHAT DO THE RESULTS OF STUDY 041 MEAN?: These results help to confirm that 72 weeks of treatment with ataluren (compared with 72 weeks of placebo treatment) slows down the decline in muscle function experienced by people living with nmDMD. Results also confirmed that side effects reported were similar with ataluren and placebo. These results likely show that people who receive ataluren can maintain their independence for a longer time. This study is the largest phase 3 clinical study of people living with nmDMD that has been done so far. NCT03179631.