Table 1 Baseline characteristics of enrolled patients.

From: Switch from enzyme replacement therapy to oral chaperone migalastat for treating fabry disease: real-life data

ID

GLA mutation

α-Gal A activity (µmol/L/h)

Age at baseline (years)

Sex

Previous ERT

ERT duration (months)

Reason for switch

1

c.1066C>T

0.5

66

M

Agalsidase beta

12

AE (nausea, flushing)

2

c.902G>C

3

20

M

Agalsidase alfa

12

AE (fever, (flushing)

3

c.901C>G

0.8

27

M

Agalsidase alfa

12

Patient choice

4

c.902G>C

0.8

22

M

Agalsidase alfa

13

Patient choice

5

c.337T>C

0.5

76

M

Agalsidase alfa

12

Patient choice

6

c.901C>G

0.5

16

M

Agalsidase alfa

20

Patient choice

7

c.901C>G

0.5

38

M

Agalsidase alfa

12

Patient choice

  1. α-Gal A α-galactosidase A, AE adverse event, ERT enzyme replacement therapy, M male.
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