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Drisapersen associated with elevated serum factor VIII levels in Duchenne muscular dystrophy

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Duchenne muscular dystrophy (DMD) is a degenerative muscle disease with over 65% of patients having a deletion of one or more exons within the DMD gene.1 Exon deletions that disrupt… Click to show full abstract

Duchenne muscular dystrophy (DMD) is a degenerative muscle disease with over 65% of patients having a deletion of one or more exons within the DMD gene.1 Exon deletions that disrupt the reading frame cause rapid mRNA degradation with no functional dystrophin protein produced.

Keywords: drisapersen associated; muscular dystrophy; elevated serum; associated elevated; duchenne muscular

Journal Title: Neurology
Year Published: 2020

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