One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better?
Sign Up to like & getrecommendations! Published in 2018 at "Therapeutic Advances in Neurological Disorders"
DOI: 10.1177/1756286418809588
Abstract: Background: Ataluren was approved for the treatment of nmDMD, both the efficacy and safety have been previously reported only from clinical trials but no report exists about real-life experience. Patient/methods: we describe three Italian children… read more here.
Keywords: age; treatment; year; three italian ... See more keywords