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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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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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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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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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The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK

Orphanet journal of rare diseases, 2008-09, Vol.3 (1), p.24-24, Article 24

2008

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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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Assessment of sleep in children with mucopolysaccharidosis type III

PloS one, 2014-02, Vol.9 (2), p.e84128-e84128

2014

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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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Development and testing of new screening method for keratan sulfate in mucopolysaccharidosis IVA

Pediatric research, 2004-04, Vol.55 (4), p.592-597

2004

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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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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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Laronidase treatment of mucopolysaccharidosis I

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

2005

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Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning

Blood, 2013-05, Vol.121 (19), p.3981-3987

2013

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Mucopolysaccharidoses and the Eye

Survey of ophthalmology, 2006, Vol.51 (1), p.1-17

2006

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Anthony H. Futerman, Ari Zimran: Gaucher Disease: Taylor & Francis, Hardcover, 2006, 528 pages, ISBN: 978-0-8493-3816-8

Human genetics, 2007-06, Vol.121 (5), p.643-644

Wraith, Ed

2007

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New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat

Therapeutics and clinical risk management, 2009, Vol.5 (default), p.877-887

2009

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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

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

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

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

The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK

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

Assessment of sleep in children with mucopolysaccharidosis type III

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

Development and testing of new screening method for keratan sulfate in mucopolysaccharidosis IVA

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

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

Laronidase treatment of mucopolysaccharidosis I

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning

Mucopolysaccharidoses and the Eye

Anthony H. Futerman, Ari Zimran: Gaucher Disease: Taylor & Francis, Hardcover, 2006, 528 pages, ISBN: 978-0-8493-3816-8

New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat

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