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Expert Opinion on Investigational Drugs Volume 12, 2003 - Issue 2
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Drug Evaluation

Substrate reduction therapy: miglustat as a remedy for symptomatic patients with Gaucher disease type 1

Gregory M Pastores Neurogenetics Unit, Department of Neurology and Pediatrics, New York University School of Medicine, New York, USA. [email protected]
&
Natalie L Barnett Neurogenetics Unit, Department of Neurology and Pediatrics, New York University School of Medicine, New York, USA. [email protected]
Pages 273-281 | Published online: 02 Mar 2005

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Terry D Butters. (2007) Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders. Expert Opinion on Pharmacotherapy 8:4, pages 427-435.
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Gregory M Pastores & Natalie L Barnett. (2005) Current and emerging therapies for the lysosomal storage disorders. Expert Opinion on Emerging Drugs 10:4, pages 891-902.
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Gregory M Pastores. (2003) Enzyme therapy for the lysosomal storage disorders: principles, patents, practice and prospects. Expert Opinion on Therapeutic Patents 13:8, pages 1157-1172.
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Brigitte A. Rigat, Michael B. Tropak, Justin Buttner, Ellen Crushell, Daphne Benedict, John W. Callahan, Douglas R. Martin & Don J. Mahuran. (2012) Evaluation of N-nonyl-deoxygalactonojirimycin as a pharmacological chaperone for human GM1 gangliosidosis leads to identification of a feline model suitable for testing enzyme enhancement therapy. Molecular Genetics and Metabolism 107:1-2, pages 203-212.
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Gregory M. Pastores. 2012. Swaiman's Pediatric Neurology. Swaiman's Pediatric Neurology 403 451 .
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Anton I. Rosenbaum & Frederick R. Maxfield. (2011) Niemann-Pick type C disease: molecular mechanisms and potential therapeutic approaches. Journal of Neurochemistry 116:5, pages 789-795.
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Gregory M. Pastores. 2011. Neurochemical Mechanisms in Disease. Neurochemical Mechanisms in Disease 785 797 .
Marc N. Offman, Marcin Krol, Israel Silman, Joel L. Sussman & Anthony H. Futerman. (2010) Molecular Basis of Reduced Glucosylceramidase Activity in the Most Common Gaucher Disease Mutant, N370S. Journal of Biological Chemistry 285:53, pages 42105-42114.
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M Rajappa, A Goyal & J Kaur. (2009) Inherited metabolic disorders involving the eye: a clinico-biochemical perspective. Eye 24:4, pages 507-518.
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Michael B. Tropak, Gregory J. Kornhaber, Brigitte A. Rigat, Gustavo H. Maegawa, Justin D. Buttner, Jan E. Blanchard, Cecilia Murphy, Steven J. Tuske, Stephen J. Coales, Yoshitomo Hamuro, Eric D. Brown & Don J. Mahuran. (2008) Identification of Pharmacological Chaperones for Gaucher Disease and Characterization of Their Effects on β-Glucocerebrosidase by Hydrogen/Deuterium Exchange Mass Spectrometry. ChemBioChem 9:16, pages 2650-2662.
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Frances M Platt & Mylvaganam Jeyakumar. (2008) Substrate reduction therapy. Acta Paediatrica 97:s457, pages 88-93.
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Gregory M Pastores. (2008) Therapeutic options for childhood-onset lysosomal storage disorders. Pediatric Health 2:1, pages 21-32.
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Paul L. M. van Giersbergen & Jasper Dingemanse. (2007) Influence of Food Intake on the Pharmacokinetics of Miglustat, an Inhibitor of Glucosylceramide Synthase. The Journal of Clinical Pharmacology 47:10, pages 1277-1282.
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Paul J. Orchard, Bruce R. Blazar, John Wagner, Lawrence Charnas, William Krivit & Jakub Tolar. (2007) Hematopoietic Cell Therapy for Metabolic Disease. The Journal of Pediatrics 151:4, pages 340-346.
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Boris Brumshtein, Harry M. Greenblatt, Terry D. Butters, Yoseph Shaaltiel, David Aviezer, Israel Silman, Anthony H. Futerman & Joel L. Sussman. (2007) Crystal Structures of Complexes of N-Butyl- and N-Nonyl-Deoxynojirimycin Bound to Acid β-Glucosidase. Journal of Biological Chemistry 282:39, pages 29052-29058.
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Mark E Haskins. (2007) Animal models for mucopolysaccharidosis disorders and their clinical relevance. Acta Paediatrica 96, pages 56-62.
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Michael Beck. (2006) New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy. Human Genetics 121:1, pages 1-22.
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Andrew A. Voss, Ana Díez-Sampedro, Bruce A. Hirayama, Donald D. F. Loo & Ernest M. Wright. (2007) Imino Sugars Are Potent Agonists of the Human Glucose Sensor SGLT3. Molecular Pharmacology 71:2, pages 628-634.
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Anna Diaz-Font, Amparo Chabás, Daniel Grinberg & Lluïsa Vilageliu. (2006) RNAi-mediated inhibition of the glucosylceramide synthase (GCS) gene: A preliminary study towards a therapeutic strategy for Gaucher disease and other glycosphingolipid storage diseases. Blood Cells, Molecules, and Diseases 37:3, pages 197-203.
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Charlotte M. Walden, Terry D. Butters, Raymond A. Dwek, Frances M. Platt & Aarnoud C. van der Spoel. (2006) Long-term non-hormonal male contraception in mice using N-butyldeoxynojirimycin. Human Reproduction 21:5, pages 1309-1315.
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Alan Milligan, Derralynn Hughes, Sian Goodwin, Linda Richfield & Atul Mehta. (2006) Intravenous enzyme replacement therapy: better in home or hospital?. British Journal of Nursing 15:6, pages 330-333.
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. 2006. Atlas of Genetic Diagnosis and Counseling. Atlas of Genetic Diagnosis and Counseling 446 451 .
Joe T. R. Clarke. 2009. A Clinical Guide to Inherited Metabolic Diseases. A Clinical Guide to Inherited Metabolic Diseases 297 323 .
Neal J. Weinreb, John A. Barranger, Joel Charrow, Gregory A. Grabowski, Henry J. Mankin & Pramod Mistry. (2005) Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. American Journal of Hematology 80:3, pages 223-229.
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Terry D. Butters, Raymond A. Dwek & Frances M. Platt. (2005) Imino sugar inhibitors for treating the lysosomal glycosphingolipidoses. Glycobiology 15:10, pages 43R-52R.
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Mylvaganam Jeyakumar, Raymond A. Dwek, Terry D. Butters & Frances M. Platt. (2005) Storage solutions: treating lysosomal disorders of the brain. Nature Reviews Neuroscience 6:9, pages 713-725.
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Ryota Suganuma, Charlotte M. Walden, Terry D. Butters, Frances M. Platt, Raymond A. Dwek, Ryuzo Yanagimachi & Aarnoud C. van der Spoel. (2005) Alkylated Imino Sugars, Reversible Male Infertility-Inducing Agents, Do Not Affect the Genetic Integrity of Male Mouse Germ Cells During Short-Term Treatment Despite Induction of Sperm Deformities1. Biology of Reproduction 72:4, pages 805-813.
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Gregory M Pastores & Patrick A Meere. (2005) Musculoskeletal complications associated with lysosomal storage disorders: Gaucher disease and Hurler-Scheie syndrome (mucopolysaccharidosis type I). Current Opinion in Rheumatology 17:1, pages 70-78.
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D.P Germain. (2004) La maladie de Gaucher : aspects cliniques, génétiques et thérapeutiques. Pathologie Biologie 52:6, pages 343-350.
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Yong-Yu Liu, Tie Yan Han, Jing Yuan Yu, Arie Bitterman, Ahn Le, Armando E. Giuliano & Myles C. Cabot. (2004) Oligonucleotides blocking glucosylceramide synthase expression selectively reverse drug resistance in cancer cells. Journal of Lipid Research 45:5, pages 933-940.
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N. Matthew Ellinwood, Charles H. Vite & Mark E. Haskins. (2004) Gene therapy for lysosomal storage diseases: the lessons and promise of animal models. The Journal of Gene Medicine 6:5, pages 481-506.
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DP Germain. (2004) Gaucher's disease: a paradigm for interventional genetics. Clinical Genetics 65:2, pages 77-86.
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Paul L McCormack & Karen L Goa. (2003) Miglustat. Drugs 63:22, pages 2427-2434.
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