Non-dystrophic myotonia: 2-year clinical and patient reported outcomes.
Timothy R FullamSwathy ChandrashekharConstantine FarmakidisOmar JawdatMamatha PasnoorMazen M DimachkieJeffrey M Statlandnull nullPublished 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.