Real world long-term outcomes in patients with mucopolysaccharidosis type II: A retrospective cohort study
Author:
Funder
Ministry of Health, Labour and Welfare
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology
Reference18 articles.
1. Epidemiology of mucopolysaccharidoses;Khan;Mol. Genet. Metab.,2017
2. Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS);Muenzer;Orphanet J. Rare Dis.,2017
3. Hematopoietic stem cell transplantation for patients with mucopolysaccharidosis II;Kubaski;Biol. Blood Marrow Transplant.,2017
4. Survival in idursulfase-treated and untreated patients with mucopolysaccharidosis type II: data from the hunter outcome survey (HOS);Burton;J. Inherit. Metab. Dis.,2017
5. Safety and efficacy of idursulfase in the treatment of mucopolysaccharidosis II (Hunter syndrome): a post-marketing study in Japan;Ueda;Expert Opin. Drug Saf.,2020
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1. An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II;Molecular Therapy;2024-03
2. Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II;Molecular Therapy - Methods & Clinical Development;2023-12
3. Results of 14-year-long Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type II: Clinical Case;Current Pediatrics;2023-01-21
4. Efficacy of early haematopoietic stem cell transplantation versus enzyme replacement therapy on neurological progression in severe Hunter syndrome: Case report of siblings and literature review;Molecular Genetics and Metabolism Reports;2022-09
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