Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome

Author:

Grewal Satkiran S.,Wynn Robert,Abdenur Jose E.,Burton Barbara K.,Gharib Maged,Haase Claudia,Hayashi Robert J.,Shenoy Shalini,Sillence David,Tiller George E.,Dudek Martha E.,van Royen-Kerkhof Annet,Wraith James E.,Woodard Paul,Young Guy A.,Wulffraat Nico,Whitley Chester B.,Peters Charles

Publisher

Elsevier BV

Subject

Genetics (clinical)

Reference12 articles.

1. Neufeld E, Muenzer J. The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D, eds. The metabolic and molecular bases of inherited disease. 8th Ed. New York: McGraw-Hill, 2001; 3421–3452.

2. Orchard PJ, Milla CE, Grewal SS, Braunlin EA, Defor TE, Krivit W et al. Determination of high-risk factors in allogeneic transplantation for Hurler syndrome [abstract]. WORLD Lysosomal diseases research network symposium 2004: 20.

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