Evaluation of treatment benefit for Inotersen within patient subgroups
FAP) disease stage
FAMILIAL AMYLOID POLYNEUROPATHY
Treatment differences in least-squares (LS) mean change in NIS and NIS-LL scores from baseline to week 65 were examined for the FAS, as well as within patient subgroups defined by the following clinical characteristics: genetic mutation (V30M, non-V30M), familial amyloid polyneuropathy (FAP) disease stage (Stage 1 [ambulatory without assistance], Stage 2 [ambulatory with assistance of cane or walker]) [The magnitude of treatment effects was also assessed using effect sizes for standardized mean differences, expressed as Cohen’s 