Table 1.

Spearman's rank correlation coefficient correlations between the MFM32 and target anchor.

Anchor	Description of scale and clinical relevance	Correlation at baseline with the MFM32	Correlation at Week 52 with the MFM32	Correlation of change from baseline to Week 52 with the MFM32
CGI-C	Single item assessing change in the patient's overall health from baseline, rated by clinicians at Week 52. Response options range from very much improved (1) to very much worse (7).	n/a	n/a	−0.48 (n = 159)
RULM	The RULM assesses the motor performance of the upper limbs in SMA. It consists of 19 scoreable items that test proximal and distal motor functions of the arm in patients with SMA (23). Higher scores indicate better motor function ability.	0.85 (n = 171)	0.87 (n = 162)	0.50 (n = 162)
SMAIS-ULM CG	The SMAIS-ULM was developed specifically for SMA in order to assess function-related independence (24). The SMAIS-ULM total score consists of 22 items focused on upper-limb-related ADLs, with higher scores indicating greater independence.	0.69 (n = 171)	0.70 (n = 163)	0.22 (n = 161)
EQ-5D-5L CG self-care item	The EQ-5D-5L is a generic self- or caregiver-reported health status questionnaire that is used to calculate a health utility score for use in health economic analysis (25). The self-care item is scored from 1 (I have no problems washing or dressing myself) to 5 (I am unable to wash or dress myself) scale.	−0.51 (n = 168)	−0.64 (n = 167)	−0.20 (n = 162)

MFM32, 32-item Motor Function Measure; CGI-C, Clinical Global Impression of Change Scale; RULM, Revised Upper Limb Module; SMA, spinal muscular atrophy; SMAIS-ULM CG, SMA Independence Scale Upper Limb Module Caregiver Report; ADLs, activities of daily living; EQ-5D-5L CG, EuroQol 5D-5L Caregiver Report.