Table 2.

Published Clinical Trials -->^* of Nusinersen^13,15–17

	SMA-1, Phase 213	SMA-2/-3, Phase 116	SMA-1, Phase 3 ENDEAR15	SMA-2/-3, Phase 3 CHErisH17
Design	Open label, dose escalation	Open label, dose escalation	Randomized, double blind, sham controlled	Randomized, double blind, sham controlled
No. of patients	20	28	121 (80 nusinersen; 41 control)	126 (84 nusinersen; 42 control)
Patients characteristics	3 wk–7 mo	2–14 yr	<7 mo	Symptom onset after 6 mo Treatment onset at 2–12 years of age
Dose	6 mg, 12 mg	1, 3, 6, 9 mg	12-mg equivalent	12 mg
Efficacy measures†	HINE-2, CHOP INTEND	HFMSE, Pediatric QoL	HINE-2	HFMSE

CHErisH, Choosing Healthy Eating for Infant Health; CHOP INTEND, Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders; ENDEAR, Efficacy and Safety of Nusinersen in Infants With Spinal Muscular Atrophy; HFMSE, Hammersmith Functional Motor Scale–Expanded; HINE-2, Hammersmith Infant Neurological Exam–Part 2; QoL, quality of life; SMA, spinal muscular atrophy

^* All 4 trials were funded by Ionis Pharmaceuticals and/or Biogen (Cambridge, MA).

^† See Table 5 for additional information about these tests.