Recombinant human alpha-glucosidase from rabbit milk in Pompe patients

H Van den Hout; A J Reuser; A G Vulto; M C Loonen; A Cromme-Dijkhuis; A T Van der Ploeg

doi:10.1016/s0140-6736(00)02533-2

Recombinant human alpha-glucosidase from rabbit milk in Pompe patients

H Van den Hout
, A J Reuser
, A G Vulto
, M C Loonen
, A Cromme-Dijkhuis
, A T Van der Ploeg^*

^*Corresponding author for this work

Research output: Contribution to journal › Article › Academic › peer-review

345 Citations (Scopus)

Abstract

Pompe's disease is a fatal muscular disorder caused by lysosomal alpha-glucosidase deficiency. In an open-label study, four babies with characteristic cardiomyopathy were treated with recombinant human alpha-glucosidase (rhGAA) from rabbit milk at starting doses of 15 mg/kg or 20 mg/kg, and later 40 mg/kg. The enzyme was generally well tolerated. Activity of alpha-glucosidase normalised in muscle. Tissue morphology and motor and cardiac function improved. The left-ventricular-mass index decreased significantly. We recommend early treatment. Long-term effects are being studied.

Original language	English
Pages (from-to)	397-398
Number of pages	2
Journal	Lancet (London, England)
Volume	356
Issue number	9227
DOIs	https://doi.org/10.1016/s0140-6736(00)02533-2
Publication status	Published - 29 Jul 2000

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This output contributes to the following UN Sustainable Development Goals (SDGs)

SDG 3 Good Health and Well-being

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10.1016/s0140-6736(00)02533-2

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title = "Recombinant human alpha-glucosidase from rabbit milk in Pompe patients",

abstract = "Pompe's disease is a fatal muscular disorder caused by lysosomal alpha-glucosidase deficiency. In an open-label study, four babies with characteristic cardiomyopathy were treated with recombinant human alpha-glucosidase (rhGAA) from rabbit milk at starting doses of 15 mg/kg or 20 mg/kg, and later 40 mg/kg. The enzyme was generally well tolerated. Activity of alpha-glucosidase normalised in muscle. Tissue morphology and motor and cardiac function improved. The left-ventricular-mass index decreased significantly. We recommend early treatment. Long-term effects are being studied.",

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T1 - Recombinant human alpha-glucosidase from rabbit milk in Pompe patients

AU - Van den Hout, H

AU - Reuser, A J

AU - Vulto, A G

AU - Loonen, M C

AU - Cromme-Dijkhuis, A

AU - Van der Ploeg, A T

PY - 2000/7/29

Y1 - 2000/7/29

N2 - Pompe's disease is a fatal muscular disorder caused by lysosomal alpha-glucosidase deficiency. In an open-label study, four babies with characteristic cardiomyopathy were treated with recombinant human alpha-glucosidase (rhGAA) from rabbit milk at starting doses of 15 mg/kg or 20 mg/kg, and later 40 mg/kg. The enzyme was generally well tolerated. Activity of alpha-glucosidase normalised in muscle. Tissue morphology and motor and cardiac function improved. The left-ventricular-mass index decreased significantly. We recommend early treatment. Long-term effects are being studied.

AB - Pompe's disease is a fatal muscular disorder caused by lysosomal alpha-glucosidase deficiency. In an open-label study, four babies with characteristic cardiomyopathy were treated with recombinant human alpha-glucosidase (rhGAA) from rabbit milk at starting doses of 15 mg/kg or 20 mg/kg, and later 40 mg/kg. The enzyme was generally well tolerated. Activity of alpha-glucosidase normalised in muscle. Tissue morphology and motor and cardiac function improved. The left-ventricular-mass index decreased significantly. We recommend early treatment. Long-term effects are being studied.

U2 - 10.1016/s0140-6736(00)02533-2

DO - 10.1016/s0140-6736(00)02533-2

M3 - Article

C2 - 10972374

SN - 0140-6736

VL - 356

SP - 397

EP - 398

JO - Lancet (London, England)

JF - Lancet (London, England)

IS - 9227

ER -

Recombinant human alpha-glucosidase from rabbit milk in Pompe patients

Abstract

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