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Non-dystrophic myotonia: 2-year clinical and patient reported outcomes.

Timothy R FullamSwathy ChandrashekharConstantine FarmakidisOmar JawdatMamatha PasnoorMazen M DimachkieJeffrey M Statlandnull null
Published in: Muscle & nerve (2022)
Symptom frequency and clinical outcome assessments were stable with defined variability in myotonia measures supporting trial designs like cross over or combined n-of-1 as important for rare disorders.
Keyphrases
  • patient reported outcomes
  • study protocol
  • clinical trial
  • phase iii
  • phase ii
  • finite element analysis
  • placebo controlled