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REVIEW

Gauchers Disease—A Reappraisal of Hematopoietic Stem Cell Transplantation

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Pages 61-70 | Received 20 Dec 2012, Accepted 20 Dec 2012, Published online: 30 Jan 2013

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Joshua L. Bonkowsky, Jacob Wilkes & David C. Shyr. (2018) Scope and Burden of Non–Standard of Care Hematopoietic Stem Cell Transplantation in Pediatric Leukodystrophy Patients. Journal of Child Neurology 33:14, pages 882-887.
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Lulu Kang, Xia Zhan, Xuefan Gu & Huiwen Zhang. (2017) Successful newborn screening for Gaucher disease using fluorometric assay in China. Journal of Human Genetics 62:8, pages 763-768.
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John Marshall, Ying Sun, Dinesh S Bangari, Eva Budman, Hyejung Park, Jennifer B Nietupski, Amy Allaire, Mary A Cromwell, Bing Wang, Gregory A Grabowski, John P Leonard & Seng H Cheng. (2016) CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease. Molecular Therapy 24:6, pages 1019-1029.
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Timothy M. Cox. (2015) Innovative Treatments for Lysosomal Diseases. Best Practice & Research Clinical Endocrinology & Metabolism 29:2, pages 275-311.
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Karin Weiss, Ashley N. Gonzalez, Grisel Lopez, Leah Pedoeim, Catherine Groden & Ellen Sidransky. (2015) The clinical management of type 2 Gaucher disease. Molecular Genetics and Metabolism 114:2, pages 110-122.
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Carla E. M. Hollak & Frits A. Wijburg. (2014) Treatment of lysosomal storage disorders: successes and challenges. Journal of Inherited Metabolic Disease 37:4, pages 587-598.
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Maciej Machaczka. (2013) Co hematolog powinien wiedzieć o chorobie Gauchera. Acta Haematologica Polonica 44:3, pages 301-306.
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