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. 2023 Jun 2;10(6):998. doi: 10.3390/children10060998

Table 1.

Patient demographics of SMA children (n = 16) treated with onasemnogene abeparvovec.

ID Sex Earlier Treatment (Age in Years), Type of Treatment Onasemnogene Abeparvovec Age (Years) SMA Type SMN2 Gene Copies Ambulation Status at the Time of Imaging Age at Imaging (Years)
1 F 0.2, Nusinersen 0.2 1 2 non-ambulatory 1.6
2 F 0.1, Nusinersen 0.2 1 2 non-ambulatory 1.4
3 M 0.2, Risdiplam 0.3 1 2 non-ambulatory 1.1
4 F 0.3 1 2 non-ambulatory 1.5
5 F 0.7 1 2 non-ambulatory 1.6
6 F 0.5, Nusinersen 0.9 1 2 non-ambulatory 2.2
7 M 0.5, Nusinersem 1.3 1 2 non-ambulatory 2.3
8 M 0.4, Nusinersen 1.3 1 2 non-ambulatory 3.7
9 M 0.3, Nusinersen 1.4 1 2 non-ambulatory 3.5
10 M 1.5 2 3 non-ambulatory 3.6
11 M 1.3, Nusinersen 1.5 2 3 minimal ambulatory 4.0
12 M -, Risdiplam 1.5 1 3 ambulatory 1.6
13 M 0.2, Nusinersen 1.7 1 2 non-ambulatory 5.6
14 M 1.6, Nusinersen 3.3 2 3 non-ambulatory 4.8
15 F 3.7 1 2 non-ambulatory 4.3
16 F 4.9 2 3 non-ambulatory 6.9