Table 3.

Baseline characteristics used for weighting in MAIC analyses in SMA

CHOP-INTEND Children’s Hospital of Philadelphia Infant Test of Neurological Disorders, HFMSE Hammersmith Function Motor Scale-Expanded, MAIC matching-adjusted indirect comparison, RULM revised upper limb module, SMA spinal muscular atrophy, SMN2 survival motor neuron 2 gene

^aDisease duration at baseline was used in lieu of age at symptom onset

^bAge at screening is not needed if age at first dose/age at symptom onset are included in MAIC weights

^cSee Table S4 Supplementary Material for how these variables were defined differently across trials

^dBaseline HFMSE or RULM score is only relevant for analyses of patients with later-onset SMA

^eAll analyses for infantile-onset included patients with 2 SMN2 copies