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Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease

PloS one, 2010-12, Vol.5 (12), p.e14192-e14192

2010

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Study of Telomere Length Reveals Rapid Aging of Human Marrow Stromal Cells following In Vitro Expansion

Stem cells (Dayton, Ohio), 2004-09, Vol.22 (5), p.675-682

2004

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Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB

PloS one, 2012-04, Vol.7 (4), p.e35787-e35787

2012

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A small proportion of mesenchymal stem cells strongly expresses functionally active CXCR4 receptor capable of promoting migration to bone marrow

Blood, 2004-11, Vol.104 (9), p.2643-2645

2004

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Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler’s syndrome after pharmacological enzyme replacement therapy

Haematologica (Roma), 2012-09, Vol.97 (9), p.1320-1328

2012

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Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy

The Journal of pediatrics, 2009-04, Vol.154 (4), p.609-611

2009

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Circadian rhythm and suprachiasmatic nucleus alterations in the mouse model of mucopolysaccharidosis IIIB

Behavioural brain research, 2010-06, Vol.209 (2), p.212-220

2010

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Signal one and two blockade are both critical for non-myeloablative murine HSCT across a major histocompatibility complex barrier

PloS one, 2013-10, Vol.8 (10), p.e77632

2013

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Clinical presentation and follow-up of patients with the attenuated phenotype of mucopolysaccharidosis type I

Acta pædiatrica (Oslo), 2005-07, Vol.94 (7), p.872-877

2005

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Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases

Journal of inherited metabolic disease, 2011-04, Vol.34 (2), p.499-508

2011

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High dose genistein in Sanfilippo syndrome: A randomised controlled trial

Journal of inherited metabolic disease, 2021-09, Vol.44 (5), p.1248-1262

2021

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Enzyme Replacement Therapy for Mucopolysaccharidoses: Opinions of Patients and Families

The Journal of pediatrics, 2008-05, Vol.152 (5), p.723-727

2008

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Thoracolumbar Kyphosis in Treated Mucopolysaccharidosis 1 (Hurler Syndrome)

Spine (Philadelphia, Pa. 1976), 2014-03, Vol.39 (5), p.381-387

2014

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Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice

Molecular genetics and metabolism, 2009-11, Vol.98 (3), p.235-242

2009

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Evaluation of heparin cofactor II–thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice

Molecular genetics and metabolism, 2010-03, Vol.99 (3), p.269-274

2010

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Retrovirally mediated correction of bone marrow–derived mesenchymal stem cells from patients with mucopolysaccharidosis type I

Blood, 2002-03, Vol.99 (5), p.1857-1859

2002

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Refsum disease is caused by mutations in the phytanoyl-CoA hydroxylase gene

Nature genetics, 1997-10, Vol.17 (2), p.190-193

1997

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Enzyme Replacement Therapy and Extended Newborn Screening for Mucopolysaccharidoses: Opinions of Treating Physicians

JIMD Reports - Case and Research Reports, 2011/1, 2011-01, Vol.1, p.9-15

2011

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Laronidase treatment of mucopolysaccharidosis I

BioDrugs : clinical immunotherapeutics, biopharmaceuticals, and gene therapy, 2005, Vol.19 (1), p.1-7

2005

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Limitations of enzyme replacement therapy: Current and future

Journal of inherited metabolic disease, 2006-04, Vol.29 (2-3), p.442-447

Wraith, J. E.

2006

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Variant Gaucher disease characterized by dysmorphic features, absence of cardiovascular involvement, laryngospasm, and compound heterozygosity for a novel mutation (D409H/C16S)

American journal of medical genetics, 2002-05, Vol.109 (4), p.328-331

2002

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Effect of discontinuing of laronidase in a patient with mucopolysaccharidosis type I

Journal of inherited metabolic disease, 2006-02, Vol.29 (1), p.230-231

2006

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The majority of MPS I patients tested raise inhibitory allo-antibodies against enzyme replacement therapy

Molecular genetics and metabolism, 2013-02, Vol.108 (2), p.S81-S81

2013

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Excess Heparan Sulphate Inhibits CXCL12-Mediated Hematopoietic Cell Migration and Engraftment After Bone Marrow Transplant in Mice with Mucopolysaccharidosis Type I

Blood, 2011-11, Vol.118 (21), p.4010-4010

2011

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Clinical presentation and follow-up of patients with the attenuated phenotype of mucopolysaccharidosis type I

Acta Paediatrica, 2005-07, Vol.94 (7), p.872-877

2005

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Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease

Study of Telomere Length Reveals Rapid Aging of Human Marrow Stromal Cells following In Vitro Expansion

Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB

A small proportion of mesenchymal stem cells strongly expresses functionally active CXCR4 receptor capable of promoting migration to bone marrow

Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler’s syndrome after pharmacological enzyme replacement therapy

Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy

Circadian rhythm and suprachiasmatic nucleus alterations in the mouse model of mucopolysaccharidosis IIIB

Signal one and two blockade are both critical for non-myeloablative murine HSCT across a major histocompatibility complex barrier

Clinical presentation and follow-up of patients with the attenuated phenotype of mucopolysaccharidosis type I

Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases

High dose genistein in Sanfilippo syndrome: A randomised controlled trial

Enzyme Replacement Therapy for Mucopolysaccharidoses: Opinions of Patients and Families

Thoracolumbar Kyphosis in Treated Mucopolysaccharidosis 1 (Hurler Syndrome)

Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice

Evaluation of heparin cofactor II–thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice

Retrovirally mediated correction of bone marrow–derived mesenchymal stem cells from patients with mucopolysaccharidosis type I

Refsum disease is caused by mutations in the phytanoyl-CoA hydroxylase gene

Enzyme Replacement Therapy and Extended Newborn Screening for Mucopolysaccharidoses: Opinions of Treating Physicians

Laronidase treatment of mucopolysaccharidosis I

Limitations of enzyme replacement therapy: Current and future

Variant Gaucher disease characterized by dysmorphic features, absence of cardiovascular involvement, laryngospasm, and compound heterozygosity for a novel mutation (D409H/C16S)

Effect of discontinuing of laronidase in a patient with mucopolysaccharidosis type I

The majority of MPS I patients tested raise inhibitory allo-antibodies against enzyme replacement therapy

Excess Heparan Sulphate Inhibits CXCL12-Mediated Hematopoietic Cell Migration and Engraftment After Bone Marrow Transplant in Mice with Mucopolysaccharidosis Type I

Clinical presentation and follow-up of patients with the attenuated phenotype of mucopolysaccharidosis type I

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