Why the study?
The study aimed to report 36-month longitudinal changes in 6MWT in ambulant patients with Duchenne muscular dystrophy amenable to skip exons 44, 45, 51, or 53.
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Ambulant DMD patients with mutations amenable to skip exon 53 experience a faster decline in 6-minute walk test distance over 36 months compared to those amenable to skip exons 44, 45, or 51, highlighting the need for genotype-specific natural history data for clinical trial design.
Brogna et al. (2019) studied this question.
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