| |
Kishnani (2009)
|
Nicolino (2009)
|
Erasmus MC
|
|---|
|
N
|
18
|
21
|
20
|
|
Deaths
|
5 (28%)
|
6 (29%)
|
4 (20%)
|
|
Age at end study in months [range]
|
34.5 [19.7-44.0]
|
41.0 [7.7-80.3]
|
60.9 [3.2-178.8]
|
|
Patients using ventilation
|
9 (50%)
|
7 (33%)
|
5 (25%)b
|
|
Dosage (every other week)
| | | |
|
15 mg/kg
|
0
|
0
|
2c, d
|
|
20 mg/kg
|
9
|
21a
|
8e
|
|
40 mg/kg
|
9
|
0
|
10d
|
- aEight patients switched to 40 mg/kg after 26 weeks due to clinical deterioration; bTwo patients died; cBoth patients switched to 30 mg/kg and later to 40 mg/kg; dEvery week; eFive patients switched to 40 mg/kg.
