Nature Medicine | Spoonful of Medicine

Researchers question ‘read-through’ mechanism of muscular dystrophy drug ataluren

A drug in clinical trials for muscular dystrophy and cystic fibrosis might not work through the molecular mechanism that scientists think it does. Although the new findings do not cast doubt on the clinical efficacy of the medication, the new experiments suggest that researchers might need to double-check that therapeutics believed to rescue normal protein production by helping parts of the cell ‘read through’ the genetic sequences actually do just that. 

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