SMA type 1 (n=6) | SMA type 2 (n=12) | SMA type 3 (n=8) | ||||
CHOP-INTEND | RULM | CHOP-INTEND | RULM | HMFSE | HMFSE | |
Baseline n, median (25%–75%) | 5, 27.5 (24.0–36.0) | 1, 9.0 | 1, 32.0 | 9, 8.5 (6.0–14.5) | 3, 32.0 (29.0–45.0) | 8, 45.0 (31.0–47.0) |
12 months n, median (25%–75%) | 5, 44.0 (38.5–55.5) | 1, 10.0 | 1, 41.0 | 9, 9.0 (7.0–13.0) | 3, 34.0 (33.0–51.0) | 8, 49.0 (40.0–52.0) |
Peripheral muscle function responders, n | 5 | 1 | 1 | 7 | 2 | 6 |
CHOP INTEND, Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders; HFMSE, Hammersmith Functional Motor Scale—Expanded; RULM, Revised Upper Limb Module.