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Statistical analysis was performed by t -test (unpaired or paired, as indicated), one-way analysis of variance (ANOVA), two-way ANOVA (time treatment or genotype treatment) with post hoc comparisons (where appropriate, as indicated in the figure legends)
Not a single case
In the Phase 2 trial, the cohorts that escalated to higher maintenance doses began at 2mg weekly and increased by 2mg every 4 weeks
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