A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy.
Erik LandfeldtThomas SejersenMár TuliniusPublished in: Acta paediatrica (Oslo, Norway : 1992) (2018)
Our clinical recommendations and proposed implementation model will inform the optimum medical management of nmDMD in Sweden and help ensure timely, equal access to ataluren and similar orphan drugs.