Table 6

Muscle function scores

SMA type 1 (n=6)SMA type 2 (n=12)SMA type 3 (n=8)
CHOP-INTENDRULMCHOP-INTENDRULMHMFSEHMFSE
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, n511726
  • CHOP INTEND, Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders; HFMSE, Hammersmith Functional Motor Scale—Expanded; RULM, Revised Upper Limb Module.