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Table 1 Key patient demographics and characteristics: ANCHOVY and FIREFISH Part 2

From: Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study

 

ANCHOVY patients

FIREFISH Part 2 patients

(N = 60)

(N = 41)

Age at SMA symptom onset, months, median (range)

1.6 (1.0–3.0)

1.5 (1.0–3.0)

Sex, n (%)

Male

39 (65)

19 (46)

Female

21 (35)

22 (54)

Country, n (%)

Belgium

5 (8)

0

Brazil

6 (10)

3 (7)

China

0

11 (27)

Croatia

3 (5)

1 (2)

France

10 (17)

4 (10)

Italy

10 (17)

10 (24)

Japan

7 (12)

1 (2)

Poland

4 (7)

4 (10)

Russia

8 (13)

5 (12)

Turkey

0

1 (2)

USA

7 (12)

1 (2)

Confirmed SMN2 copy number, n (%)

Two

30 (50)

41 (100)*

Unknown

30 (50)

0

  1. *Patients in FIREFISH were required to have two copies of SMN2 per the inclusion criteria
  2. SMA, spinal muscular atrophy; SMN2, survival of motor neuron 2