Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients

Robert F. Wynn; Jean Mercer; Joanne Page; Trevor F. Carr; Simon Jones; J. Edmond Wraith

doi:10.1016/j.jpeds.2008.07.004

Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients

Robert F. Wynn, Jean Mercer, Joanne Page, Trevor F. Carr, Simon Jones, J. Edmond Wraith

Research output: Contribution to journal › Article › peer-review

Abstract

We describe the use of enzyme replacement therapy in conjunction with hematopoietic stem cell transplantation in 18 consecutive patients with severe mucopolysaccharidosis type I. The survival and engraftment rate was 89% overall and 93% for the 15 patients who received full-intensity conditioning. © 2009 Mosby, Inc. All rights reserved.

Original language	English
Pages (from-to)	135-139
Number of pages	4
Journal	Journal Of Pediatrics
Volume	154
Issue number	1
DOIs	https://doi.org/10.1016/j.jpeds.2008.07.004
Publication status	Published - Jan 2009

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title = "Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients",

abstract = "We describe the use of enzyme replacement therapy in conjunction with hematopoietic stem cell transplantation in 18 consecutive patients with severe mucopolysaccharidosis type I. The survival and engraftment rate was 89\% overall and 93\% for the 15 patients who received full-intensity conditioning. {\textcopyright} 2009 Mosby, Inc. All rights reserved.",

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AU - Jones, Simon

AU - Wraith, J. Edmond

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AB - We describe the use of enzyme replacement therapy in conjunction with hematopoietic stem cell transplantation in 18 consecutive patients with severe mucopolysaccharidosis type I. The survival and engraftment rate was 89% overall and 93% for the 15 patients who received full-intensity conditioning. © 2009 Mosby, Inc. All rights reserved.

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Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients

Abstract

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