A favorable outcome in an infantile-onset Pompe patient with cross reactive immunological material (CRIM) negative disease with high dose enzyme replacement therapy and adjusted immunomodulation-Reference-Cited by-同舟云学术

A favorable outcome in an infantile-onset Pompe patient with cross reactive immunological material (CRIM) negative disease with high dose enzyme replacement therapy and adjusted immunomodulation

Published:2022-09 Issue: Volume:32 Page:100893
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Curelaru Shiri,Desai Ankit K.,Fink Daniel,Zehavi Yoav,Kishnani Priya S.,Spiegel Ronen

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference26 articles.

1. Glycogen storage disease type II: Acid α-glucosidase (acid maltase) deficiency;Hirschhorn,2001

2. The natural course of infantile Pompe’s disease: 20 original cases compared with 133 cases from the literature;van den Hout;Pediatrics,2003

3. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants;Kishnani;Mol. Genet. Metab.,2010

4. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease;Kishnani;Neurology.,2007

5. Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease;Kishnani;Pediatr. Res.,2009

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1. Subject: Comment on Spataro et al.;Pediatric Allergy and Immunology;2024-08

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4. Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy;Frontiers in Immunology;2024-04-23

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