Responsiveness of outcome measures in myotonic dystrophy type 1.
Kirsten L KnakAisha M SheikhNanna WittingJohn VissingPublished in: Annals of clinical and translational neurology (2020)
Overall, adequate responsiveness was shown for both muscle strength dynamometers, TUG and mCTSIB. These outcomes are therefore likely candidate endpoints for clinical trials lasting 1 year. Most static balance tests are not responsive and not recommended in a heterogeneous DM1 population.