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1.Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres

作者：Arunabha Ghosh^a ; ^c ; ^{arunabha.ghosh@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author} ; Weston Miller^b ; ^{mill4991@umn.edu" class="auth_mail" title="E-mail the corresponding author} ; Paul J. Orchard^b ; ^{orcha001@umn.edu" class="auth_mail" title="E-mail the corresponding author} ; Simon A. Jones^a ; ^{simon.jones@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author} ; Jean Mercer^a ; ^{jean.mercer@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author} ; Heather J. Church^a ; ^{heather.church@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author} ; Karen Tylee^a ; ^{karen.tylee@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author} ; Troy Lund^b ; ^{lundx072@umn.edu" class="auth_mail" title="E-mail the corresponding author} ; Brian W. Bigger^c ; ^{brian.bigger@manchester.ac.uk" class="auth_mail" title="E-mail the corresponding author} ; Jakub Tolar^b ; ^{tolar003@umn.edu" class="auth_mail" title="E-mail the corresponding author} ; Robert F. Wynn^d ; ^{robert.wynn@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author}

关键词：EFS ; event-free survival ; ERT ; enzyme replacement therapy ; GAG ; glycosaminoglycan ; GvHD ; graft versus host disease ; HSCT ; haematopoietic stem cell transplantation ; LVEF ; left ventricular ejection fraction ; MPS I ; Mucopolysaccharidosis Type I ; OS ; overall survival ; STR ; short tandem repeats ; VNTR ; variable number tandem repeats

刊名：Molecular Genetics and Metabolism

出版年：2016

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