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Table 1 Demographics and clinical characteristics

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

CharacteristicTreatment-evaluable patients (N = 56)
n (%)
Gender49 (87.5)a
Female29 (59.2)
Male20 (40.8)
SMN2 copy number53 (94.6)a
12 (3.8)
230 (56.5)
315 (28.3)
46 (11.3)
>40
SMA type56 (100)a
Presymptomatic5 (8.9)
I38 (67.8)
II8 (14.3)
III5 (8.9)
Age at first treatment43 (76.8)a
0–6 months26 (60.5)
> 6–24 months12 (27.9)
> 24 months5 (11.6)
  1. aPercentage based on eligible patients, n/N (%)