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Correspondence
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Volume 360:194-195 January 8, 2009 Number 2
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Elimination of Antibodies to Recombinant Enzyme in Pompe's Disease

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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.1,2,3 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.

A baby boy of African-American . . . [Full Text of this Article]



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