Aartsma-Rus A, Arechavala-Gomeza V. Why dystrophin quantification is key in the eteplirsen saga. Nature reviews. Neurology 2018;14:454-456.
Eteplirsen, a compound designed to restore dystrophin in patients with Duchenne muscular dystrophy , controversially received approval by the FDA in 2016. Owing to limited clinical data, the approval was based on eteplirsen’s effect on dystrophin expression. Now , the dystrophin quantification results have been published, and although low levels of dystrophin expression are shown, the quantification remains debatable.