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BioMarin Provides Update on GALNS Phase 1/2 Extension Study (MOR-100)
Date:7/28/2011

>

37.6

16

14.6

(65.2)

18.9

15

13.9

(98.6)

14.0

15

21.8

(85.4)

15.7

14

29.1

(79.1)

6.86 Minute Walk Test – change from Baseline (meters)

(for patients with baseline walk distance < 325m)

N

Mean

(Std. Dev.)

Median

12

38.0

(45.2)

42.4

12

28.2

(61.0)

24.4

11

37.3

(79.8)

14.0

11

40.0

(77.0)

15.7

11

38.3

(81.8)

2.63 Minute Stair Climb Test – change from Baseline (steps/minute)

N

Mean

(Std. Dev.)

Median

15

6.9

(8.9)

7.3

15

8.9

(14.3)

10.2

14

14.5

(14.3)

13.1

14

13.9

(15.2)

14.6

12

13.6

(19.6)

10.4PULMONARY FUNCTIONFVC – Percent Change

from BaselineN

Mean

(Std. Dev.)

Median

15

1.5

(16.2)

0.0

15

7.2

(16.0)

10.0

14

11.5

(15.5)

6.9N/A

13

15.9

(16.8)

12.0MVV – Percent Change

from Baseline

N

Mean

(Std. Dev.)

Median

13

11.0

(21.5)

6.4

14

10.5

(17.4)

10.0

13

11.1

(16.4)

10.6N/A

12

10.3

(23.2)

0.8*   After Week 36 in MOR-002, patients were dosed at 1.0 mg/kg/week for 36 to 48 weeks.  At Week 0 of MOR-100, patients were returned to 2.0 mg/kg/week dosing.

** Results were impacted by several patients with extenuating circumstances (knee procedures, car accident, unplanned extended drug holiday) after week 24 of MOR-002.About BioMarinBioMarin develops and commercializes innovative biopharmaceuticals for se
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SOURCE BioMarin Pharmaceutical Inc.
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