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

Table 1 Demographics and clinical characteristics

Characteristic	Treatment-evaluable patients (N = 56) n (%)
Gender	49 (87.5)^a
Female	29 (59.2)
Male	20 (40.8)
SMN2 copy number	53 (94.6)^a
1	2 (3.8)
2	30 (56.5)
3	15 (28.3)
4	6 (11.3)
>4	0
SMA type	56 (100)^a
Presymptomatic	5 (8.9)
I	38 (67.8)
II	8 (14.3)
III	5 (8.9)
Age at first treatment	43 (76.8)^a
0–6 months	26 (60.5)
> 6–24 months	12 (27.9)
> 24 months	5 (11.6)

ISSN: 1750-1172