Table 2.

Individual real-world endpoints from baseline to the 24-month follow-up after gene replacement therapy. Patients were first sorted from left to right by SMN2 copy number, second by SMA type, and third by age at gene transfer therapy infusion. A green circle  represents the acquisition of a developmental milestone (i.e., head holding, sitting for 30 seconds unassisted or standing for a few seconds with aid). A red circle represents the necessity of medical support (i.e., gastrostomy, NIV or spinal brace). An empty box represents the absence of developmental milestones or the need for medical support. The dashed boxes indicate that the data are not available at M24 (insufficient follow-up time). NIV stands for sleep NIV (i.e., <16 hr/day). The two patients who died are not represented. NIV: noninvasive ventilation, SMA: spinal muscular atrophy.