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A qualitative study on the impact of caring for an ambulatory individual with nonsense mutation Duchenne muscular dystrophy

10 Aug 2021

Abstract excerpt

BACKGROUND: Duchenne muscular dystrophy is a rare genetic neuromuscular disorder, which can result in early death due to disease progression. Ataluren is indicated for the treatment of nonsense mutation Duchenne muscular dystrophy, in ambulatory individuals aged two years and older. This study explored the impact of caring for an ambulatory individual with nonsense mutation Duchenne muscular dystrophy, as well as...

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PMID: 34374872
DOI: 10.1186/s41687-021-00344-8

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