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Table 2 SMA symptoms at diagnosis of treated patients

From: 10th European Conference on Rare Diseases & Orphan Products (ECRD 2020)

 Treatment received
Symptom, n (%)Onasemnogene abeparvovec → nusinersen (n = 2)Nusinersen → onasemnogene abeparvovec (n = 17)Nusinersen → onasemnogene abeparvovec → nusinersen (n = 8)Onasemnogene abeparvovec only (n = 18)Nusinersen only (n = 11)All treated patients (N = 56)
Patients with symptoms, n2168151051
Hypotonia1 (50.0)12 (75.0)8 (100.0)13 (86.7)7 (70.0)41 (80.4)
Limb weakness2 (100.0)13 (81.3)7 (87.5)11 (73.3)9 (90.0)42 (82.4)
Pneumonia or respiratory symptoms2 (100.0)10 (62.5)5 (62.5)5 (33.3)1 (10.0)23 (45.1)
Tongue fasciculations010 (62.5)3 (37.5)6 (40.0)019 (37.3)
Developmental delay1 (50.0)8 (50.0)5 (62.5)8 (53.3)5 (50.0)27 (52.9)
Constipation1 (50.0)5 (31.3)4 (50.0)2 (13.3)4 (40.0)16 (31.4)
Swallowing or feeding difficulties1 (50.0)12 (75.0)7 (87.5)8 (53.3)1 (10.0)29 (56.9)
Other00002 (20.0)2 (3.9)