Table 1:

Demographics and Clinical Information from 48 Study Participants. Abbreviations: W= white; B= black; A= Asian; Unk= unknown; Non H/L= not Hispanic or Latino; H/L= Hispanic or Latino; del= deletion; dupl= duplication; n/a = not available; TTSTAND=time to stand test from the floor; m/s=meters per second

Dose Level (mg/ kg)	Age at Enrollment (years)	Race	Ethnicity	Mutation type	Starting exon	Baseline TTSTAND Velocity (m/s)	Baseline TTSTAND Seconds
0.25	6	W	Non H/L	Multiple Exon Del	8	0.13	7.85
	4	W	Non H/L	Multiple Exon Del	8	0.18	5.53
	4	W	Non H/L	Multiple Exon Del	47	0.26	3.9
	6	B	Non H/L	Multiple Exon Del	51	0.13	7.59
	6	W	Non H/L	No large del/dupl	n/a	0.14	7.3
	6	W	Non H/L	Single Exon Del	45	0.2	5
	6	W	Non H/L	No large del/dupl	n/a	0.1	10.2
	6	W	Non H/L	No large del/dupl	n/a	0.27	3.65
	4	W	Non H/L	Multiple Exon Del	8	0.16	6.44
	4	W	Non H/L	Multiple Exon Del	45	0.13	7.8
	4	W	Non H/L	Multiple Exon Del	10	0.29	3.4
	6	W	Non H/L	Multiple Exon Del	52	0.22	4.5
0.75	6	A	Non H/L	Multiple Exon Del	8	0.14	7.14
	4	W	Non H/L	Single Exon Del	44	0.25	4.02
	4	W	Non H/L	Multiple Exon Del	46	0.12	8.4
	5	Unk	Hisp or Lat	Single Exon Del	45	0.13	7.8
	5	W	Non H/L	Multiple Exon Del	45	0.13	7.97
	4	W	Non H/L	Multiple Exon Del	5	0.33	3.06
	4	W	Non H/L	Multiple Exon Del	51	0.33	3
	4	W	Non H/L	Multiple Exon Del	16	0.23	4.31
	6	W	Non H/L	Multiple Exon Del	48	0.27	3.7
	5	W	Non H/L	Multiple Exon Del	49	0.31	3.2
	6	W	Non H/L	Multiple Exon Del	49	0.38	2.8
	5	W	Non H/L	Single Exon Del	51	0.27	3.7
2	4	W	Non H/L	Multiple Exon Del	22	0.28	3.6
	4	W	Non H/L	No large del/dupl	n/a	0.18	5.7
	4	W	Non H/L	Multiple Exon Del	33	0.17	6.03
	4	W	Non H/L	Single Exon Del	50	0.12	8.5
	4	W	Non H/L	Multiple Exon Del	53	0.16	6.1
	6	W	Non H/L	Multiple Exon Del	3	0.26	3.8
	4	W	Non H/L	Multiple Exon Del	45	0.11	9.2
	5	W	Non H/L	Multiple Exon Del	48	0.26	3.9
	6	W	Non H/L	Multiple Exon Del	3	0.21	4.7
	5	W	Non H/L	No large del/dupl	n/a	0.29	3.5
	6	W	Non H/L	Multiple Exon Del	8	0.26	3.9
	4	W	Non H/L	Single Exon Del	8	0.4	2.5
6	5	W	Hisp or Lat	Multiple Exon Del	48	0.15	6.5
	5	W	Hisp or Lat	Single Exon Del	45	0.16	6.4
	5	W	Non H/L	Multiple Exon Del	44	0.14	7
	5	W	Non H/L	Multiple Exon Del	48	0.25	4
	6	W	Non H/L	Single Exon Del	51	0.24	4.1
	4	W	Non H/L	Single Exon Del	52	0.07	14.06
	4	W	Non H/L	Single Exon Dupl	12	0.26	3.81
	4	W	Non H/L	Single Exon Del	7	0.21	4.7
	4	W	Non H/L	Multiple Exon Del	48	0.16	6.15
	4	W	Non H/L	Single Exon Del	44	0.23	4.31
	6	W	Non H/L	No large del/dupl	n/a	0.2	5
	5	W	Non H/L	Single Exon Del	45	0.21	4.8