Literature DB >> 19712183

Combined enzyme replacement and haematopoietic stem cell transplantation in Hurler syndrome.

Sunita Bijarnia1, Peter Shaw, Anne Vimpani, Robert Smith, Verity Pacey, Helen O'Grady, John Christodoulou, David Sillence.   

Abstract

We report the long-term follow-up of successful treatment of mucopolysaccharidosis type I H (MPS IH, Hurler syndrome) with combined enzyme replacement therapy and haematopoietic progenitor stem cell transplant.

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Year:  2009        PMID： 19712183     DOI： 10.1111/j.1440-1754.2009.01537.x

Source DB:  PubMed          Journal:  J Paediatr Child Health        ISSN： 1034-4810            Impact factor:   1.954


  4 in total

1.  A Dysmorphometric Analysis to Investigate Facial Phenotypic Signatures as a Foundation for Non-invasive Monitoring of Lysosomal Storage Disorders.

Authors:  Stefanie Kung; Mark Walters; Peter Claes; Jack Goldblatt; Peter Le Souef; Gareth Baynam
Journal:  JIMD Rep       Date:  2012-06-10

2.  Combined Enzyme Replacement Therapy and Hematopoietic Stem Cell Transplantation in Mucopolysacharidosis Type VI.

Authors:  D Sillence; K Waters; S Donaldson; P J Shaw; C Ellaway
Journal:  JIMD Rep       Date:  2011-09-06

3.  Long term survival and cardiopulmonary outcome in children with Hurler syndrome after haematopoietic stem cell transplantation.

Authors:  Su Han Lum; Karolina M Stepien; Arunabha Ghosh; Alexander Broomfield; Heather Church; Jean Mercer; Simon Jones; Robert Wynn
Journal:  J Inherit Metab Dis       Date:  2017-03-10       Impact factor: 4.982

4.  Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.

Authors:  Marialuisa Alliegro; Rita Ferla; Edoardo Nusco; Chiara De Leonibus; Carmine Settembre; Alberto Auricchio
Journal:  Mol Ther       Date:  2016-09-23       Impact factor: 11.454
  4 in total

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