Extended Data Table 2 Part B – Summary of statistical testing

From: High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial

  1. aENDEAR matched sham, CHERISH matched sham and CHERISH matched 12/12 mg indicate prespecified subsets of participants matched to characteristics of the DEVOTE 50/28 mg group. Comparisons to CHERISH were performed using the day 279 DEVOTE visit since this was the closest visit to the day 274 CHERISH visit.
  2. CHOP-INTEND, Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders; EFS, event-free survival; HFMSE, Hammersmith Functional Motor Scale Expanded; HINE-2, Hammersmith Infant Neurological Exam section 2; NfL, neurofilament light chain; OS, overall survival; RULM, Revised Upper Limb Module; SMA, spinal muscular atrophy.
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