Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. - Wikidata - awgldk - TriplyDB

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines.

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines.

http://www.wikidata.org/entity/Q35079584

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Extracorporeal photopheresis versus alternative treatment for chronic graft-versus-host disease after haematopoietic stem cell transplantation in paediatric patients Extracorporeal photopheresis versus standard treatment for acute graft-versus-host disease after haematopoietic stem cell transplantation in paediatric patients Extracorporeal photopheresis versus alternative treatment for chronic graft-versus-host disease after haematopoietic stem cell transplantation in paediatric patients Extracorporeal photopheresis versus standard treatment for acute graft-versus-host disease after haematopoietic stem cell transplantation in paediatric patients Extracorporeal photopheresis versus standard treatment for acute graft-versus-host disease after haematopoietic stem cell transplantation in paediatric patients Hematopoietic stem cell transplantation for Gaucher disease Extracorporeal photopheresis versus alternative treatment for chronic graft-versus-host disease after haematopoietic stem cell transplantation in paediatric patients Hematopoietic stem cell transplantation for Gaucher disease Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis Review and evaluation of the methodological quality of the existing guidelines and recommendations for inherited neurometabolic disorders CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)Orthopaedic management of Hurler's disease after hematopoietic stem cell transplantation: a systematic review.Developing therapeutic approaches for metachromatic leukodystrophy Enzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type mice Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.Optimization of immunomagnetic separation for cord blood-derived hematopoietic stem cells.Chronic graft-versus-host disease (GVHD) in children.The role of microglia in human disease: therapeutic tool or target?Hematopoietic stem cell transplantation for infantile osteopetrosis.Unrelated umbilical cord blood transplant for juvenile metachromatic leukodystrophy: a 5-year follow-up in three affected siblings.Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases Mortality in patients with morquio syndrome a.Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Molecular Genetics and Metabolism Report Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: clinical, biochemical, and pathological improvements.Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.Estimating demand and unmet need for allogeneic hematopoietic cell transplantation in the United States using geographic information systems.Clinical implications of chimerism after allogeneic hematopoietic stem cell transplantation in children with non-malignant diseases Farber disease: clinical presentation, pathogenesis and a new approach to treatment.Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report.The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I.Hematopoietic cell transplantation activity in Europe for inherited metabolic diseases: open issues and future directions.Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation Current and emerging therapies for the lysosomal storage disorders.Haemopoietic stem cell transplantation for genetic disorders.Determinants of health care use in a population-based leukodystrophy cohort.Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?Juvenile metachromatic leukodystrophy: understanding the disease and implications for nursing care.Novel candidate disease for gene therapy: metachromatic leukodystrophy.Umbilical cord blood transplantation for juvenile metachromatic leukodystrophy.

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Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines.

http://www.wikidata.org/entity/Q35079584

description

2003 nî lūn-bûn

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2003 թուականի Փետրուարին հրատարակուած գիտական յօդուած

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2003 թվականի փետրվարին հրատարակված գիտական հոդված

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2003年の論文

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Hematopoietic cell transplanta ...... comes and practice guidelines.

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Bone Marrow Transplantation

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Hematopoietic cell transplanta ...... comes and practice guidelines.

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C Peters

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International Bone Marrow Transplant Registry

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National Marrow Donor Program

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Working Party on Inborn Errors, European Bone Marrow Transplant Group

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P2860

Defects in TCIRG1 subunit of the vacuolar proton pump are responsible for a subset of human autosomal recessive osteopetrosis

Mutations in the a3 subunit of the vacuolar H(+)-ATPase cause infantile malignant osteopetrosis

Loss of the ClC-7 chloride channel leads to osteopetrosis in mice and man

Subunits of the translation initiation factor eIF2B are mutant in leukoencephalopathy with vanishing white matter

Carbonic anhydrase II deficiency in 12 families with the autosomal recessive syndrome of osteopetrosis with renal tubular acidosis and cerebral calcification

Successful bone-marrow transplantation for infantile malignant osteopetrosis.

Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and S

The defect in Hurler and Hunter syndromes. II. Deficiency of specific factors involved in mucopolysaccharide degradation

Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres.

The importance of testing for adrenoleucodystrophy in males with idiopathic Addison's disease

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229-239

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10.1038/SJ.BMT.1703839

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4

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31

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10870508

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1028761877

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12621457

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