10
views
0
recommends
+1 Recommend
0 collections
    0
    shares
      • Record: found
      • Abstract: found
      • Article: not found

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

      Lancet
      Animals, Animals, Genetically Modified, Cardiomyopathy, Hypertrophic, complications, drug therapy, Glycogen Storage Disease Type II, Humans, Milk, chemistry, Rabbits, Recombinant Proteins, isolation & purification, therapeutic use, alpha-Glucosidases

      Read this article at

      ScienceOpenPublisherPubMed
      Bookmark
          There is no author summary for this article yet. Authors can add summaries to their articles on ScienceOpen to make them more accessible to a non-specialist audience.

          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.

          Related collections

          Author and article information

          Comments

          Comment on this article