Transforming the clinical outcome in CRIM-negative infantile Pompe disease identified via newborn screening: the benefits of early treatment with enzyme replacement therapy and immune tolerance induction
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Title
Transforming the clinical outcome in CRIM-negative infantile Pompe disease identified via newborn screening: the benefits of early treatment with enzyme replacement therapy and immune tolerance induction
Authors
Keywords
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Journal
GENETICS IN MEDICINE
Volume -, Issue -, Pages -
Publisher
Springer Science and Business Media LLC
Online
2021-01-26
DOI
10.1038/s41436-020-01080-y
References
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Related references
Note: Only part of the references are listed.- Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature
- (2020) Aleena A. Khan et al. GENETICS IN MEDICINE
- Benefits of Prophylactic Short-Course Immune Tolerance Induction in Patients With Infantile Pompe Disease: Demonstration of Long-Term Safety and Efficacy in an Expanded Cohort
- (2020) Ankit K. Desai et al. Frontiers in Immunology
- Earlier and higher dosing of alglucosidase alfa improve outcomes in patients with infantile-onset Pompe disease: Evidence from real-world experiences
- (2020) Yin-Hsiu Chien et al. Molecular Genetics and Metabolism Reports
- Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy
- (2018) Rossella Parini et al. Orphanet Journal of Rare Diseases
- Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study
- (2016) C. M. van Gelder et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Very Early Treatment for Infantile-Onset Pompe Disease Contributes to Better Outcomes
- (2016) Chia-Feng Yang et al. JOURNAL OF PEDIATRICS
- CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy
- (2015) Kathryn L. Berrier et al. GENETICS IN MEDICINE
- Response of 33 UK patients with infantile-onset Pompe disease to enzyme replacement therapy
- (2015) A. Broomfield et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Long-Term Prognosis of Patients with Infantile-Onset Pompe Disease Diagnosed by Newborn Screening and Treated since Birth
- (2015) Yin-Hsiu Chien et al. JOURNAL OF PEDIATRICS
- Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease
- (2015) Laura E. Case et al. NEUROMUSCULAR DISORDERS
- Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease
- (2014) Carin M. van Gelder et al. JOURNAL OF INHERITED METABOLIC DISEASE
- 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
- (2013) Suhrad G. Banugaria et al. PLoS One
- Assessing disease severity in Pompe disease: The roles of a urinary glucose tetrasaccharide biomarker and imaging techniques
- (2012) Sarah P. Young et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART C-SEMINARS IN MEDICAL GENETICS
- Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease
- (2012) Yoav H. Messinger et al. GENETICS IN MEDICINE
- The emerging phenotype of long-term survivors with infantile Pompe disease
- (2012) Sean N. Prater et al. GENETICS IN MEDICINE
- 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
- 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
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