Elimination of Antibodies to Recombinant Enzyme in Pompe's Disease
- 8 January 2009
- journal article
- letter
- Published by Massachusetts Medical Society in The New England Journal of Medicine
- Vol. 360 (2), 194-195
- https://doi.org/10.1056/nejmc0806809
Abstract
Infantile Pompe's disease is due to a deficiency of lysosomal acid alpha glucosidase (GAA). In patients in whom GAA is not produced, a status called cross-reacting immunologic material (CRIM)–negative, enzyme-replacement therapy with recombinant human GAA (rhGAA) has uniformly led to high titers of anti-rhGAA antibody, with an ultimately fatal outcome.1 Previous attempts at eliminating rhGAA antibodies in these patients have failed.1-3 We report the successful induction of immune modulation in a CRIM-negative patient with Pompe's disease who continues to be antibody-free at 24 months of age and continues to gain motor milestones.Keywords
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