Issue Date: July 1, 2013
Doubt Cast On Muscle Disorder Drug Candidate’s Mechanism
An experimental drug in human clinical trials for muscular dystrophy doesn’t work in the way scientists had originally proposed, a study finds. In 2007, PTC Therapeutics developed the small molecule ataluren as a readthrough agent, a molecule that restores protein levels in cells with mutations that block production of those proteins. In cells and in mice with a muscular dystrophy mutation, ataluren boosts levels of the disease-related protein and improves muscle function, . . .
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