Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT
Published 2013 View Full Article
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Title
Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT
Authors
Keywords
Antibodies, Enzyme replacement therapy, Immune response, Methotrexate, Immune suppression, Immune tolerance, Ventilators, Algorithms
Journal
PLoS One
Volume 8, Issue 6, Pages e67052
Publisher
Public Library of Science (PLoS)
Online
2013-06-26
DOI
10.1371/journal.pone.0067052
References
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Related references
Note: Only part of the references are listed.- CRIM-negative Pompe disease patients with satisfactory clinical outcomes on enzyme replacement therapy
- (2013) Hamoud Al Khallaf et al. MOLECULAR GENETICS AND METABOLISM
- Predicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: Lessons learned from 10 years of clinical laboratory testing experience
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- Immune modulation in Pompe disease treated with enzyme replacement therapy
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- Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease
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- Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: Need for agents to target antibody-secreting plasma cells
- (2012) Suhrad G. Banugaria et al. MOLECULAR GENETICS AND METABOLISM
- Rituximab resistance
- (2011) Andrew R. Rezvani et al. BEST PRACTICE & RESEARCH CLINICAL HAEMATOLOGY
- The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease
- (2011) Suhrad G. Banugaria et al. GENETICS IN MEDICINE
- Atypical immunologic response in a patient with CRIM-negative Pompe disease
- (2011) Mary-Alice Abbott et al. MOLECULAR GENETICS AND METABOLISM
- Intravenous gammaglobulin suppresses inflammation through a novel TH2 pathway
- (2011) Robert M. Anthony et al. NATURE
- CRIM-negative infantile Pompe disease: 42-month treatment outcome
- (2010) Marianne Rohrbach et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease
- (2009) Marc Nicolino et al. GENETICS IN MEDICINE
- Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants
- (2009) Priya S. Kishnani et al. MOLECULAR GENETICS AND METABOLISM
- Elimination of Antibodies to Recombinant Enzyme in Pompe's Disease
- (2009) Nancy J. Mendelsohn et al. NEW ENGLAND JOURNAL OF MEDICINE
- Early Treatment With Alglucosidase Alfa Prolongs Long-Term Survival of Infants With Pompe Disease
- (2009) Priya S Kishnani et al. PEDIATRIC RESEARCH
- Pompe Disease in Infants: Improving the Prognosis by Newborn Screening and Early Treatment
- (2009) Y.-H. Chien et al. PEDIATRICS
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