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

  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},
  volume={360 2},
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
Highly Cited
This paper has 96 citations. REVIEW CITATIONS


Publications citing this paper.
Showing 1-10 of 48 extracted citations

97 Citations

Citations per Year
Semantic Scholar estimates that this publication has 97 citations based on the available data.

See our FAQ for additional information.