Responsiveness of outcome measures in myotonic dystrophy type 1.

Ann Clin Transl Neurol · 2020 · PMC7448158 · PMID 32672404

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highly significantno p-value reported

Thus, based on this study, the best outcomes for clinical trials within 1 year are the stationary dynamometry or HHD with measurements of the proximal muscle groups and the balance assessments TUG or mCTSIB, because these tests capture subtle, but highly significant changes after 1 year of no intervention.

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