A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy

Landfeldt E, Sejersen T, Tulinius M. A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy. Acta paediatrica (Oslo, Norway : 1992) 2019;108:224-230   Abstract AIM: Ataluren has been approved for treating nonsense mutation Duchenne muscular dystrophy (nmDMD), and there are currently discussions concerning drug access and applications beyond the […]