Statistical analysis
EVENTS, EVENT
Safety endpoints are evaluated descriptively. The number and percentage of patients reporting AEs, and the number of AEs reported are presented. Listings with the events tabulated by system organ class and preferred term are available as supplementary material (Additional file The number of patients experiencing an AE compared descriptively between groups. All patients with AEs listed individually with subject number in addition to type of event, start and stop time, duration, seriousness, severity, action taken, relationship to trial drug and outcome of AE was presented to the members of the DSMB.Baseline characteristics of the first 20 subjects are presented as mean ± standard deviation (SD) or number (n) and fraction (%) (Table Demographic characteristics for the safety analysis cohort (N = 20)Baseline data is compared with available norm data from reference populations in Sweden for RAND-36 and EQ-5D and published international reference values for 6MWT and 30 s CST [All data for efficacy continuous endpoints at baseline are presented using mean, SD, and 95% confidence interval. Comparisons against norm data was done using unpaired t-test.No adjustment for multiplicity was done since results are descriptive. A p value < 0.05 was considered statistically significant. All reported p values are two-sided. All bar graphs are presented as mean and CI. Significant difference I presented as: *p < 0.05; **p < 0.01; ***p < 0.001.