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Table 3 α-Gal A activity in peripheral blood mononuclear cells (PBMCs, nmol/hr/mg protein)

From: Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

 

Screening

Baseline

Week 4

Week 8

Week 12

Week 16

Week 20

Week 24

FAB CL-202 (NCT00283959) 1

2-01022

0.15

0.14

0.14

0.18

0.12

-

-

-

2-0103

0.22

0.24

2.23

1.76

2.3

-

-

-

2-0104

0.5

0.21

2.88

3.18

3.36

-

-

-

2-0202

0.5

0.3

7.53

6.45

7.12

-

-

-

FAB CL-203 (NCT00283933) 1

3-03012

0.13

0.08

0.17

0.17

0.22

0.19

0.25

0.36

3-0302

0.18

0.10

0.08

0.07

BLQ

0.06

0.08

0.13

3-0303

BLQ

0.14

0.36

0.37

BLQ

0.22

0.42

0.3

3-RF01

6.11

2.6

8.46

12.1

10.6

11.5

8.35

10.9

3-RF03

0.04

0.2

0.3

0.57

0.38

0.51

0.34

1.32

  1. 1 Study number.
  2. 2 Patient ID.
  3. BLQ = Below limit of quantification. Enzyme activity was reported as nmol/hour/mg protein.