TABLE 1.

Demographics, genetics, and treatment characteristics of SMA subjects

Subject	SMA type	Sex	Age at first dose	Number of nusinersen doses received	Copy number of SMN1 exon 8 gene	Copy number of SMN2 exon 7 gene	Copy number of SMN2 exon 8 gene	Copy number of NAIP exon 5 gene	CSF sample	CHOP INTEND or HFMSE
SMA003	1	F	6 months	15	0	2	2	0	+	+
SMA005	1	M	10 years	7	0	2	2	0	+	HFMS
SMA007	1	M	7 months	7	0	2	2	n.a.	+	HFMS
SMA008	1	M	4 months	7	0	2	2	0	+	+
SMA012	1	M	7 months	11	0	2	2	0	+	+
SMA013	1	F	3 years	13	0	3	3	2	+	+
SMA014	1	M	3 months	10	0	2	2	1	+	+
SMA015	1	M	4 months	10	0	2	2	1	+	+
SMA016	1	M	8 years	7	0	2	2	0	+	+
SMA018	1	F	3 years	14	0	2	2	0	+	+
SMA022	1	M	5 years	9	0	2	2	0	+	+
SMA026	1	M	8 years	6	0	3	3	1	+	+
SMA033	1	M	1 months	6	1	2	1	1	+	HFMS
SMA035	1	F	9 years	7	0	2	2	0	n.a.	+
SMA021	1	F	3 years	10	1	2	2	1	n.a.	+
SMA037	1	M	6 years	5	0	2	2	0	n.a.	+
SMA002	2	M	8 years	12	0	3	3	1	+	+
SMA024	2	M	3 years	5	0	3	3	n.a.	+	HFMS
SMA029	2	F	12 years	9	1	1	1	1	+	+
SMA030	2	M	1 years	7	1	3	2	1	+	+
SMA001	3	F	17 years	8	0	4	4	2	+	+
SMA004	3	F	13 years	12	1	3	3	2	+	+
SMA006	3	M	14 years	13	1	3	3	2	+	+
SMA009	3a	M	10 years	12	1	2	2	1	+	+
SMA010	3	M	15 years	12	1	4	3	2	+	+
SMA017	3a	F	5 years	11	0	3	3	2	+	+
SMA019	3b	F	8 years	12	0	3	4	2	+	+
SMA028	3a	F	4 years	11	0	2	3	0	+	+
SMA031	3b	M	12 years	10	0	4	4	2	+	+
SMA036	3b	M	17 years	7	0	3	3	2	n.a.	+

Abbreviations: CHOP INTEND, Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders; F, Female; HFMS, Hammersmith Functional Motor Scale; HFMSE, Hammersmith Functional Motor Scale Expanded; M, Male; m, months; n.a., not available; NAIP, NLR Family Apoptosis Inhibitory Protein; SMA, spinal muscular atrophy; SMN, survival motor neuron; y, years.