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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.
AuthorsH Van den Hout, A J Reuser, A G Vulto, M C Loonen, A Cromme-Dijkhuis, A T Van der Ploeg
JournalLancet (London, England) (Lancet) Vol. 356 Issue 9227 Pg. 397-8 (Jul 29 2000) ISSN: 0140-6736 [Print] England
PMID10972374 (Publication Type: Letter, Research Support, Non-U.S. Gov't)
Chemical References
  • Recombinant Proteins
  • alpha-Glucosidases
Topics
  • Animals
  • Animals, Genetically Modified
  • Cardiomyopathy, Hypertrophic (complications, drug therapy)
  • Glycogen Storage Disease Type II (complications, drug therapy)
  • Humans
  • Milk (chemistry)
  • Rabbits
  • Recombinant Proteins (isolation & purification, therapeutic use)
  • alpha-Glucosidases (isolation & purification, therapeutic use)

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