Figure 5

Neuromuscular Symptom Score (NSS) in both groups in 18 patients (sequence deflazacort-placebo, and placebo-deflazacort, completer population). a) Average course%NSS sum score from natural history until end of both sequences. b) Mutation-related NSS subgroup analysis. Correlation between the type of mutation, resulting in residual dysferlin protein versus total loss of dysferlin protein, assessed by Western Blot and mutation prediction, and outcomes during treatment phases with a sample size of 4 patients in 3 subgroups (sequence deflazacort-placebo, protein present / sequence deflazacort-placebo, protein absent / sequence placebo-deflazacort, protein present) and 6 patients in the remaining subgroup (sequence placebo-deflazacort, protein absent).