Ataluren is a small-molecule protein restoration therapy compound in clinical development for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation (nmDMD), cystic fibrosis caused by a nonsense mutation (nmCF), and other …
Ataluren is a small-molecule protein restoration therapy compound in clinical development for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation (nmDMD), cystic fibrosis caused by a nonsense mutation (nmCF), and other …
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