Table 1 Dose range, plasma Phe, and pregnancy in pegvaliase-treated PKU female mice

From: Maternal phenylketonuria syndrome: studies in mice suggest a potential approach to a continuing problem

Study

No. of dams

Avg. age at treatment start (weeks)

Age at mating (weeks)

Male genotype

Time on treatment while mated (weeks)

Avg. phe when mated at 4 PM (μM)

08-005a

8

10.0 (1.2b)

21.8 (2.4)

PKU

11.7 (3.2)

150 (20)

08-027c

8

6.1 (0.3)

32.3 (6.3)

HET

26.1 (6.4)

230 (70)

09-068ad

12

5.7 (3.1)

16.7 (4/3)

HET

11.0 (2.3)

340 (80)

09-68be

12

29.4 (4.3)f

29.4 (4.3)

HET

0.3 (0)g

140 (30)

Study

No. of litters

Avg. age of litter (weeks)

Weaned pups

No. of pups lost

   

♂ +/−

♀ +/−

♂ −/−

♀ −/−

 

08-005

2

23.1 (1.9)

—

—

3

5

 

08-027

6

38.9 (8.1)

8

8

5

13

2

09-068a

20

23.4 (5.3)

26

34

30

24

6

09-068b

6

32.0 (3.8)

10

2

5

6

 

Study

No. of NNMh

Age (avg.) at NNM

Est. no. of pupsi

Born livej

08-005

7

30.3 (3.8)

48

 

08-027

19

43.7 (8.2)

73

 

09-068a

19

26.3 (6.6)

65

18

09-068b

9

37.4 (4.2)

31

3

  1. F, Friday; HET, heterozygous; M, Monday; NNM, neonatal mortality; Phe, phenylalanine; PKU, phenylketonuria; W, Wednesday.
  2. a40 mg/kg; M, F (4 PM).
  3. bStandard deviation (sample).
  4. c10, 10, and 20 mg/kg; M, W, F (4 PM).
  5. d10 mg/kg; M (8 AM), W, F (4 PM).
  6. e6 mg/kg; daily (4 PM).
  7. fTransfer from 09-068a, last dose 3/24/09.
  8. gBegin new dose schedule 3/26/09.
  9. hNNM: Pup loss/death within 24 h of parturition.
  10. iNumber of pups determined by weight loss divided by 2 g per pup and/or counting any remains if possible. NNM assumed if weight loss and blood/debris in cage.
  11. jAlive at birth, dead, or missing next day.
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