Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease | Zendy

Yoav H. Messinger | Zendy; Nancy J. Mendelsohn | Zendy; William J. Rhead | Zendy; David Dimmock | Zendy; Eli Hershkovitz | Zendy; Michael Champion | Zendy; Simon Jones | Zendy; Rebecca Olson | Zendy; Amy White | Zendy; Cara Wells | Zendy; Deeksha Bali | Zendy; Laura E. Case | Zendy; Sarah P. Young | Zendy; Amy S. Rosenberg | Zendy; Priya S. Kishnani | Zendy

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Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease

Author(s) -

Yoav H. Messinger,

Nancy J. Mendelsohn,

William J. Rhead,

David Dimmock,

Eli Hershkovitz,

Michael Champion,

Simon Jones,

Rebecca Olson,

Amy White,

Cara Wells,

Deeksha Bali,

Laura E. Case,

Sarah P. Young,

Amy S. Rosenberg,

Priya S. Kishnani

Publication year - 2012

Publication title -

genetics in medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 3.509

H-Index - 128

eISSN - 1530-0366

pISSN - 1098-3600

DOI - 10.1038/gim.2011.4

Subject(s) - enzyme replacement therapy , rituximab , antibody , medicine , methotrexate , immune tolerance , immune system , immunology , disease

Infantile Pompe disease resulting from a deficiency of lysosomal acid α-glucosidase (GAA) requires enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA). Cross-reactive immunologic material negative (CRIM-negative) Pompe patients develop high-titer antibody to the rhGAA and do poorly. We describe successful tolerance induction in CRIM-negative patients.

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