Abstract
Exondys 51 (eteplirsen) is approved in the US to treat patients (pts) with Duchenne Muscular Dystrophy (DMD) who have a confirmed mutation of the DMD gene amenable to exon 51 skipping. The rarity of DMD makes it difficult to assess real-world outcomes in these pts. This study uses a data set with claims covering a majority of the US population and linked electronic medical records (EMR) for a subset to assess health and resource utilization outcomes in treated and untreated DMD pts. Linked administrative claims and EMR data (2011-2019) from Decision Resources Group were used.
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