Combined enzyme replacement and haematopoietic stem cell transplantation in Hurler syndrome
Author:
Publisher
Wiley
Subject
Pediatrics, Perinatology and Child Health
Link
http://onlinelibrary.wiley.com/wol1/doi/10.1111/j.1440-1754.2009.01537.x/fullpdf
Reference11 articles.
1. Physical performance testing in mucopolysaccharidosis I: a pilot study;Dumas;Pediatr. Rehabil.,2004
2. Prevalence of lysosomal storage disorders;Meikle;JAMA,1999
3. Can mucopolysaccharidosis type I disease severity be predicted based on a patient's genotype? A comprehensive review of the literature;Terlato;Genet. Med.,2003
4. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-l-iduronidase (laronidase);Wraith;J. Pediatr.,2004
5. Hurler syndrome: outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children;Peters;Blood,1998
Cited by 13 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. Enzyme replacement combinational therapy: effective treatments for mucopolysaccharidoses;Expert Opinion on Biological Therapy;2021-03-11
2. Neonatal combination therapy improves some of the clinical manifestations in the Mucopolysaccharidosis type I murine model;Molecular Genetics and Metabolism;2020-07
3. Assessments of neurocognitive and behavioral function in the mucopolysaccharidoses;Molecular Genetics and Metabolism;2017-12
4. Cognitive and adaptive measurement endpoints for clinical trials in mucopolysaccharidoses types I, II, and III: A review of the literature;Molecular Genetics and Metabolism;2017-06
5. Long term survival and cardiopulmonary outcome in children with Hurler syndrome after haematopoietic stem cell transplantation;Journal of Inherited Metabolic Disease;2017-03-10
1.学者识别学者识别
2.学术分析学术分析
3.人才评估人才评估
"同舟云学术"是以全球学者为主线，采集、加工和组织学术论文而形成的新型学术文献查询和分析系统，可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容，当前同舟云学术共收录了国内外主流学术期刊6万余种，收集的期刊论文及会议论文总量共计约1.5亿篇，并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询！咨询电话：010-8811{复制后删除}0370
www.globalauthorid.com
TOP
Copyright © 2019-2024 北京同舟云网络信息技术有限公司 京公网安备11010802033243号 京ICP备18003416号-3