Elimination of antibodies to recombinant enzyme in Pompe's disease.

@article{Mendelsohn2009EliminationOA,
  title={Elimination of antibodies to recombinant enzyme in Pompe's disease.},
  author={Nancy J. Mendelsohn and Yoav H Messinger and Amy S. Rosenberg and Priya Kishnani},
  journal={The New England journal of medicine},
  year={2009},
  volume={360 2},
  pages={
          194-5
        }
}
To the Editor: 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… CONTINUE READING
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