Table 3.

Clinical outcome measures in patients with SMA types 2 and 3 treated with nusinersen

	SMA type 2a (n = 20)	SMA type 2b (n = 10)	SMA type 3a (n = 16)
Follow-up in months	34.5 (5–44)	36 (13–40)	33.5 (21–41)
Number of injections	11.5 (4–14)	11.5 (6–15)	11 (8–13)
HFMSE score	16 (0–41)	20 (4–51)	55.5 (31–64)
△HFMSE score	5 (−3–24)	4 (−2–13)	6 (−3–15)
WHO motor milestone score	1 (0–4)	1 (1–5)	6 (2–6)
Use of ventilator support n (%)
None	15 (75)	10 (100)	16 (100)
Non-invasive ventilation	5 (25)	0 (0)	0 (0)
Hours of non-invasive ventilation	12 (12)	n.a.	n.a.
Invasive ventilation	0 (0)	0 (0)	0(0)
Dependent on feeding tube n (%)
None	16 (80)	7 (70)	16 (100)
Nasogastric tube	0 (0)	1 (10)	0 (0)
Gastrostomy	4 (20)	2 (20)	0 (0)

SMA = spinal muscular atrophy; n = number; HFMSE = Hammersmith Functional Motor Scale Expanded; △HFMSE = difference in HFMSE score between baseline and last follow-up assessment, WHO = World Health Organization.

Outcomes present the data of last follow-up assessment, unless otherwise stated.

All outcomes are given in median (range) unless otherwise stated.