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-Reference-Cited by-同舟云学术

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

Published:2022-09 Issue: Volume:32 Page:100881
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Sreekantam Srividya,Smith Laura,Stewart Catherine,Kearney Shauna,Lawson Sarah,Raiman Julian,Vijay Suresh,Santra Saikat

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference16 articles.

1. Intrafamilial Variability in the Clinical Manifestations of Mucopolysaccharidosis Type II: Data from the Hunter Outcome Survey (HOS);Ficicioglu;Am J Med Genet A.,2018

2. A phase I/II study of intrathecal Idursulfase-IT in children with severe mucopolysaccharidosis II;Muenzer;Genet. Med.,2016

3. Mutational spectrum of the iduronate-2-sulfatase (IDS) gene in 36 unrelated Russian MPS II patients;Karsten;Hum. Genet.,1998

4. Impact of enzyme replacement therapy and hematopoietic stem cell therapy on growth in patients with hunter syndrome;Patel;Mol. Genet. Metab. Rep.,2014

5. Activities of daily living in patients with hunter syndrome: impact of enzyme replacement therapy and hematopoietic stem cell transplantation;Tanjuakio;Mol. Genet. Metab.,2015

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