Improvements in health status and utility associated with ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy

Landfeldt E, Lindberg C, Sejersen T. Improvements in health status and utility associated with ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy. Muscle & nerve 2020;61:363-368   Abstract INTRODUCTION: Little is known of the impact of ataluren on health status and utility in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). In this […]