phase 1b-2 trial

Complete results of a U.K. trial of an exon-skipping drug in boys with DMD show drug is safe, increases dystrophin production

posted on July 26, 2011 - 11:24am
Complete and extremely encouraging findings from a phase 1b-2 trial of eteplirsen (AVI-4658), an exon skipping drug in development to treat a portion of the Duchenne muscular dystrophy (DMD) population, show the compound is safe and well-tolerated, and that it can significantly increase production of the needed dystrophin protein in recipients without eliciting an unwanted immune response.

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