One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better?

Ruggiero L, Iodice R, Esposito M, et al. One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better? Therapeutic advances in neurological disorders  2018;11:1756286418809588   Abstract Background: Ataluren was approved for the treatment of nmDMD, both the efficacy and safety have been previously reported only from clinical trials […]