Fig. 1: Parts B and C: patient disposition.
From: High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial
The Part B cohort with infantile-onset SMA was compared with a matched group of 20 participants from ENDEAR who had received sham. The Part B cohort with later-onset SMA was compared with two matched groups of participants from CHERISH who had received sham (n = 16) and 12/12 mg nusinersen (n = 32).
