Statistical Analysis
The sample size was determined based on the primary endpoint. With a minimum expected decrease in CTx of 60% with ALN, a non-inferiority margin of 12% (80% of efficacy retained), an SD of 15% on change in CTx, and a significance level of 5%, 54 participants should be included in the study in order to obtain statistical power of 90%. We adjusted this to 64 participants (32 in each group) to account for dropouts. Baseline variables were checked for normality using qq-plots and log-transformed as appropriate. Data are displayed as means ± SD for normally distributed data and median with CIs for non-normally distributed data. We calculated percent changes in BTMs from baseline to EOS, checked normality of data using qq-plots and compared differences between groups using independent samples 