Fig. 2. Growth charts for children with three copies of SMN2.

Ten (67%) children achieved the ability to maintain weight at or above the WHO 3rd percentile without the need for non-oral/mechanical feeding support at all visits up to 24 months of age. The ability to maintain weight at or above the 3rd percentile without the need for non-oral/mechanical feeding support was defined by meeting both of the following criteria at all visits: (1) does not receive nutrition through mechanical support (that is, feeding tube) and (2) maintains weight (≥3rd percentile for age and sex as defined by WHO guidelines) consistent with the patient’s age at the assessment. The gray shading represents WHO growth standards for the 3rd through 97th percentiles. n = 6 males and n = 9 females; mean (s.d.) age at dosing, 28.7 (11.68) days.