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April 11, 2017

Error in Text

JAMA. 2017;317(14):1482. doi:10.1001/jama.2017.2823

In the Viewpoint entitled “Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy,”1 published online October 24, 2016, and in the December 13, 2016, issue of JAMA, there were errors in the text. In the first paragraph beginning with the second sentence, the text should have read, “Duchenne muscular dystrophy is a progressive X-linked genetic disease caused by mutations in a gene that produces the protein dystrophin that helps stabilize muscle fibers. It is usually fatal by the third decade of life. No disease-modifying treatments are available.” In the second paragraph, the first 2 sentences should have read, “Eteplirsen offered a promising new therapeutic approach that would correct a mutation in a gene coding for dystrophin, allowing production of a truncated but functional version of the protein. In particular, eteplirsen was designed to skip exon 51, which would address the mutations in about 10% to 15% of patients with DMD (an estimated 2000-2500 cases in the United States).” This article was corrected online.