American Academy of Pediatrics

TABLE 1

Summary of Important Clinical and Safety Data in All Treated Children

Patient	Age at Infusion, mo	Wt at Infusion, kg	SMN2 Copy No.	Nusinersen Previously?	Platelets Day 7	AST Peak (nl <60)	ALT Peak (nl <50)	GGT Peak (nl <78)	Fever on Day 3–4	No. Weeks on “Full Steroid Dose”
1^a	1	4.2	2	No	154	62	36	—	No	4
2^a	2	5.4	3	No	—	53	53	20	No	6
3^a	3	4.8	3	No	186	92	79	38	No	4
4	4	6.5	2	No	179	124	67	23	Yes	4
5	4	5.1	2	No	234	507	469	201	Yes	11
6	4.5	5.9	2	Yes	384	53	40	20	No	6
7	5	6.5	2	No	297	129	96	58	No	4
8^a	5	9.2	4	No	204	144	89	13	No	4
9^a	6	8.8	4	Yes	219	172	192	62	Yes	11
10	8	7.5	2	Yes	33	339	228	111	Yes	10.5
11	8	7.9	2	Yes	65	122	62	17	Yes	4
12	8	8.1	2	No	171	807	1187	251	No	14
13	10	9.7	3	No	146	118	113	21	Yes	6
14	11	10	2	Yes	129	83	54	18	No	4
15	12	8.2	3	No	559^b	1471	1794	163	Yes	10
16	17	11	2	Yes	108	116	108	26	Yes	4
17	17	8.2	3	Yes	142	177	212	13	No	8
18	19	12	3	Yes	127	1040	1137	83	Yes	10
19	22	8.0	3	Yes	110	1953	1935	376	No	13.5
20	23	10.9	2	Yes	182	66	47	15	No	7
21	23	11.7	2	Yes	52	1114	1002	240	No	11

Patient	Age at Infusion, mo	Wt at Infusion, kg	SMN2 Copy No.	Nusinersen Previously?	Platelets Day 7	AST Peak (nl <60)	ALT Peak (nl <50)	GGT Peak (nl <78)	Fever on Day 3–4	No. Weeks on “Full Steroid Dose”
1^a	1	4.2	2	No	154	62	36	—	No	4
2^a	2	5.4	3	No	—	53	53	20	No	6
3^a	3	4.8	3	No	186	92	79	38	No	4
4	4	6.5	2	No	179	124	67	23	Yes	4
5	4	5.1	2	No	234	507	469	201	Yes	11
6	4.5	5.9	2	Yes	384	53	40	20	No	6
7	5	6.5	2	No	297	129	96	58	No	4
8^a	5	9.2	4	No	204	144	89	13	No	4
9^a	6	8.8	4	Yes	219	172	192	62	Yes	11
10	8	7.5	2	Yes	33	339	228	111	Yes	10.5
11	8	7.9	2	Yes	65	122	62	17	Yes	4
12	8	8.1	2	No	171	807	1187	251	No	14
13	10	9.7	3	No	146	118	113	21	Yes	6
14	11	10	2	Yes	129	83	54	18	No	4
15	12	8.2	3	No	559^b	1471	1794	163	Yes	10
16	17	11	2	Yes	108	116	108	26	Yes	4
17	17	8.2	3	Yes	142	177	212	13	No	8
18	19	12	3	Yes	127	1040	1137	83	Yes	10
19	22	8.0	3	Yes	110	1953	1935	376	No	13.5
20	23	10.9	2	Yes	182	66	47	15	No	7
21	23	11.7	2	Yes	52	1114	1002	240	No	11

—, not applicable.

Denotes those identified via newborn screening.

Denotes the child who did not have a decline in platelets. Patient 1 did not have GGT checked. Patient 2 was not able to obtain laboratories on day 7.

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