Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I-Reference-Cited by-同舟云学术

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Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I

Published:2019-06 Issue: Volume:13 Page:321-333
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Ghosh Arunabha,Liao Aiyin,O’Leary Claire,Mercer Jean,Tylee Karen,Goenka Anu,Holley Rebecca,Jones Simon A.,Bigger Brian W.

Funder

UK MPS Society

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference53 articles.

1. Mucopolysaccharidosis I: management and treatment guidelines;Muenzer;Pediatrics,2009

2. Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure;de Ru;Orphanet J. Rare Dis.,2011

3. Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients;Wynn;J. Pediatr.,2009

4. Management of mucopolysaccharidosis type IH (Hurler’s syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution’s experience;Wiseman;J. Inherit. Metab. Dis.,2013

5. Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres;Ghosh;Mol. Genet. Metab.,2016

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1. Advances in Immune Tolerance Induction in Enzyme Replacement Therapy;Pediatric Drugs;2024-04-25

2. High-Dose ERT, Rituximab, and Early HSCT in an Infant with Wolman’s Disease;New England Journal of Medicine;2024-02-15

3. Omics-Based Approaches for the Characterization of Pompe Disease Metabolic Phenotypes;Biology;2023-08-23

4. Advances in therapies for neurological lysosomal storage disorders;Journal of Inherited Metabolic Disease;2023-05-02

5. Antibodies against recombinant enzyme in the treatment of Fabry disease: Now you see them, now you don’t;Molecular Therapy - Methods & Clinical Development;2022-12

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