1. Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.
- Author
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Van den Hout, Hannerieke, Reuser, Arnold J J, Vulto, Arnold G, Loonen, M Christa B, Cromme-Dijkhuis, Adri, Van der Ploeg, Ans T, Van den Hout, H, Reuser, A J, Vulto, A G, Loonen, M C, Cromme-Dijkhuis, A, and Van der Ploeg, A T
- Subjects
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GLUCOSIDASES , *MUSCLE disease treatment , *DEFICIENCY diseases , *CARDIOMYOPATHIES , *THERAPEUTICS , *ANIMAL experimentation , *COMPARATIVE studies , *CARDIAC hypertrophy , *GLYCOSIDASES , *RESEARCH methodology , *MEDICAL cooperation , *MILK , *RABBITS , *RECOMBINANT proteins , *RESEARCH , *TRANSGENIC animals , *EVALUATION research , *INBORN errors of carbohydrate metabolism , *DISEASE complications - 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. [ABSTRACT FROM AUTHOR]
- Published
- 2000
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