Enhancing the effects of nusinersen with cybernic treatment using Hybrid Assistive Limb (HAL) in spinal muscular atrophy: a real-world case series and exploratory cohort analysis

Table 1 Patient demographics and baseline characteristics in cohorts 1 and 2

Patient identifier	Cohort 1 (plus HAL)						Cohort 2 (Conventional therapy only)
Patient identifier	NH-01	NH-02	NH-03	NH-06	TH-01	Mean (median)	NH-04	NH-05	NH-07	TH-02	TH-03	KH-01	KH-02	Mean (median)
Sex	Female	Female	Female	Male	Male		Male	Male	Male	Male	Female	Female	Male
Age, years	11	9	62	65	33	36.0 (33)	12	10	7	43	7	71	22	24.6 (12)
Age of SMA onset, years	1	1	1	19	6	5.6 (1)	1	1	1	6	1	11	6	3.7 (1)
Time from SMA onset to nusinersen administration, years	10	8.1	61.2	45.7	26.5	30.3 (26.5)	10.1	8.8	7.1	37	6	59.9	15.7	20.7 (10.1)
Type of SMA	2	2	3	3	3		3	3	2	3	2	3	3
SMN1 gene deletion	Y	Y	Y	Y	Y		Y	Y	Y	Y	Y	Y	Y
SMN2 copy numbers	2	3	4	4	3		3	3	3	3	3	4	4
Height, cm	140	116	151	181	172.2	152.0 (151)	136.1	122.2	110	182	101	141.8	165.4	136.9 (136.1)
Body weight, kg	22	28.3	48.6	72.6	79	50.1 (48.6)	29.1	21.4	15	96.5	13.5	56.9	54.9	41.0 (29.1)
Previous HAL use	Y	N	Y	Y	N		N	N	N	N	N	N	N
Time from BL to start of HAL, days	204	269	0	191	104		-	-	-	-	-	-	-
BL motor function
HFMSE total score, points	38	15	42	25	55	35.0 (38)	51	50	18	43	17	42	46	38.1 (43)
RULM total score, points	33	20	33	11	37	26.8 (33)	37	-	-	37	20	37	37	33.6 (37)
2MWT with hoist, m	30.66	8.17	26.16	37.64	102.30	35.97 (30.66)	50.75	55.50	-	47.10	-	93.00	-	61.59 (53.13)
Able to walk with hoist	Y	Y	Y	Y	Y		Y	Y	Y	Y	Y	Y	Y
Usual mobility	EWC	EWC	Handrail	EWC	Handrail		WSOO	WSOO	EWC	Cane	EWC	Cane	Handrail

2MWT, 2-minute walk test; BL, baseline; EWC, electric wheelchair; HAL, Hybrid Assistive Limb; HFMSE, Hammersmith Functional Motor Scale-Expanded; N, no; PT, physiotherapy; RULM, Revised Upper Limb Module; SMA spinal muscular atrophy; SMN, survival motor neuron (gene); WSOO, walking support outside only; Y, yes

ISSN: 1750-1172