Genistin-rich soy isoflavone extract in substrate reduction therapy for Sanfilippo syndrome: An open-label, pilot study in 10 pediatric patients.

[1]  G. Węgrzyn,et al.  Abnormalities in the hair morphology of patients with some but not all types of mucopolysaccharidoses , 2008, European Journal of Pediatrics.

[2]  R. Erickson.  A first therapy for Niemann-Pick C , 2007, The Lancet Neurology.

[3]  M. Patterson,et al.  Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study , 2007, The Lancet Neurology.

[4]  G. Węgrzyn,et al.  Rapid deterioration of a patient with mucopolysaccharidosis type I during interruption of enzyme replacement therapy , 2007, American journal of medical genetics. Part A.

[5]  M. Mcentee,et al.  Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid. , 2007, Molecular genetics and metabolism.

[6]  R. Hoft,et al.  A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years. , 2007, Molecular genetics and metabolism.

[7]  J. Fletcher,et al.  Inhibition of Glycosaminoglycan Synthesis Using Rhodamine B in a Mouse Model of Mucopolysaccharidosis Type IIIA , 2006, Pediatric Research.

[8]  C. Eng,et al.  A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome) , 2006, Genetics in Medicine.

[9]  G. Węgrzyn,et al.  Genistein-mediated inhibition of glycosaminoglycan synthesis as a basis for gene expression-targeted isoflavone therapy for mucopolysaccharidoses , 2006, European Journal of Human Genetics.

[10]  J. Wittes,et al.  Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. , 2006, The Journal of pediatrics.

[11]  W. Walop,et al.  Subacute Sclerosing Panencephalitis: Results of the Canadian Paediatric Surveillance Program and review of the literature , 2005, BMC pediatrics.

[12]  G. Węgrzyn,et al.  Changes in hair morphology of mucopolysaccharidosis I patients treated with recombinant human α‐L‐iduronidase (laronidase, Aldurazyme) , 2005, American journal of medical genetics. Part A.

[13]  F. Pfannkuch,et al.  Subchronic and chronic safety studies with genistein in dogs. , 2005, Food and chemical toxicology : an international journal published for the British Industrial Biological Research Association.

[14]  R. Giugliani,et al.  Heparan sulfate levels in mucopolysaccharidoses and mucolipidoses , 2005, Journal of Inherited Metabolic Disease.

[15]  T. Tsai.  Concurrent measurement of unbound genistein in the blood, brain and bile of anesthetized rats using microdialysis and its pharmacokinetic application. , 2005, Journal of chromatography. A.

[16]  W. Mobley,et al.  Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I. , 2004, Molecular genetics and metabolism.

[17]  G. Węgrzyn,et al.  A general model for genetic regulation of turnover of glycosaminoglycans suggests a possible procedure for prediction of severity and clinical progress of mucopolysaccharidoses. , 2004, Medical hypotheses.

[18]  Joseph Muenzer,et al.  Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). , 2004, The Journal of pediatrics.

[19]  M. Schell,et al.  Safety and pharmacokinetics of purified soy isoflavones: single-dose administration to postmenopausal women. , 2002, The American journal of clinical nutrition.

[20]  C. Scriver,et al.  The Metabolic and Molecular Bases of Inherited Disease, 8th Edition 2001 , 2001, Journal of Inherited Metabolic Disease.

[21]  J. Belmont,et al.  Enzyme-replacement therapy in mucopolysaccharidosis I. , 2001, The New England journal of medicine.

[22]  S. Barnes,et al.  Mechanisms of action of the soy isoflavone genistein: emerging role for its effects via transforming growth factor beta signaling pathways. , 1998, The American journal of clinical nutrition.

[23]  V. Hascall,et al.  Hyaluronan Synthesis by Mouse Cumulus Cells Is Regulated by Interactions between Follicle-stimulating Hormone (or Epidermal Growth Factor) and a Soluble Oocyte Factor (or Transforming Growth Factor β1)* , 1997, The Journal of Biological Chemistry.

[24]  M. Nester.  Use of a Brief Assessment Examination in a Study of Subacute Sclerosing Panencephalitis , 1996, Journal of child neurology.

[25]  M. Pisano,et al.  Epidermal growth factor potentiates the induction of ornithine decarboxylase activity by prostaglandins in embryonic palate mesenchymal cells: effects on cell proliferation and glycosaminoglycan synthesis. , 1987, Developmental biology.

[26]  M. Shibuya,et al.  Genistein, a specific inhibitor of tyrosine-specific protein kinases. , 1987, The Journal of biological chemistry.

[27]  F. Wilcoxon.  Individual Comparisons by Ranking Methods , 1945 .

[28]  G. Węgrzyn,et al.  Substrate deprivation therapy: a new hope for patients suffering from neuronopathic forms of inherited lysosomal storage diseases , 2010, Journal of Applied Genetics.

[29]  U. Ullmann,et al.  Safety, tolerability, and pharmacokinetics of single ascending doses of synthetic genistein (Bonistein™) in healthy volunteers , 2005, Advances in therapy.