TABLE 2.

Select Neurological Function Assessments Used in SMA Clinical Trials

Measure	Description
Hammersmith Infant Neurologic Exam (HINE Section 2)83,84	Used for assessing various aspects of neurologic function in infants ages 2 months to 2 years 3 sections, 26 items Section 1: Neurologic assessment Section 2: Developmental milestone assessment Section 3: Behavioral assessment Section 2 may be used alone 8 items, scores of 0 to 2, 3, or 4 Children with SMA1 may score 0 on all 8 items
Hammersmith Functional Motor Scale, Expanded (HFMSE)85,86	Used to evaluate motor function in individuals with later-onset SMA (SMA2 and SMA3) 33 items Total score ranges from 0 to 66; lower scores indicate poorer function Scores in patients with SMA2 or SMA3 may decline over 12 months
Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)87,88	Used to evaluate motor skills of children with SMA ages ~4 months to 4 years Includes 16 items to assess motor skills, each graded on a scale of 0 (no response) to 4 (complete response) Total score ranges from 0 to 64; lower scores indicate poorer function Infants with SMA may score much lower than unaffected infants A score exceeding 40 is rarely seen in infants with SMA1
Motor Function Measure–32 Item(MFM-32)89	Used to evaluate motor function in children and adults with neuromuscular diseases Assesses 32 items in 3 dimensions (standing and transfers, axial and proximal motor function, distal motor function) Total score ranges from 0 to 96; lower scores indicate poorer function

SM A = spinal muscular atrophy.