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Expert Opinion on Therapeutic Patents Volume 13, 2003 - Issue 8
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Review

Enzyme therapy for the lysosomal storage disorders: principles, patents, practice and prospects

Gregory M Pastores Neurogenetics Unit, Departments of Neurology and Pediatrics, New York University School of Medicine, New York, USA. [email protected]
Pages 1157-1172 | Published online: 02 Mar 2005

Citations (13)

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Read on this site (3)

Juan M Benito, José M García Fernández & Carmen Ortiz Mellet. (2011) Pharmacological chaperone therapy for Gaucher disease: a patent review. Expert Opinion on Therapeutic Patents 21:6, pages 885-903.
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Gregory M Pastores. (2007) Agalsidase alfa (Replagal™) in the treatment of Anderson-Fabry disease. Biologics: Targets and Therapy 1:3, pages 291-300.
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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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Articles from other publishers (10)

Pilar Alfonso, Vanesa Andreu, Almudena Pino‐Angeles, Aurelio A. Moya‐García, M. Isabel García‐Moreno, José C. Rodríguez‐Rey, Francisca Sánchez‐Jiménez, Miguel Pocoví, Carmen Ortiz Mellet, Jose M. García Fernández & Pilar Giraldo. (2013) Bicyclic Derivatives of L ‐Idonojirimycin as Pharmacological Chaperones for Neuronopathic Forms of Gaucher Disease . ChemBioChem 14:8, pages 943-949.
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M. Aguilar-Moncayo, T. Takai, K. Higaki, T. Mena-Barragán, Y. Hirano, K. Yura, L. Li, Y. Yu, H. Ninomiya, M. I. García-Moreno, S. Ishii, Y. Sakakibara, K. Ohno, E. Nanba, C. Ortiz Mellet, J. M. García Fernández & Y. Suzuki. (2012) Tuning glycosidase inhibition through aglycone interactions: pharmacological chaperones for Fabry disease and GM1 gangliosidosis. Chemical Communications 48:52, pages 6514.
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Gregory M. Pastores. 2012. Swaiman's Pediatric Neurology. Swaiman's Pediatric Neurology 403 451 .
Gregory M. Pastores. 2011. Neurochemical Mechanisms in Disease. Neurochemical Mechanisms in Disease 785 797 .
G.M. Pastores. 2009. Encyclopedia of Neuroscience. Encyclopedia of Neuroscience 571 580 .
Gregory M Pastores. (2008) Therapeutic options for childhood-onset lysosomal storage disorders. Pediatric Health 2:1, pages 21-32.
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Kerry Anne McEachern, John Fung, Svetlana Komarnitsky, Craig S. Siegel, Wei-Lien Chuang, Elizabeth Hutto, James A. Shayman, Gregory A. Grabowski, Johannes M.F.G. Aerts, Seng H. Cheng, Diane P. Copeland & John Marshall. (2007) A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease. Molecular Genetics and Metabolism 91:3, pages 259-267.
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Gregory M Pastores & Swati Sathe. (2006) A Chaperone-Mediated Approach to??Enzyme Enhancement as a Therapeutic Option for the Lysosomal??Storage Disorders. Drugs in R & D 7:6, pages 339-348.
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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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G M Pastores. (2004) Agalsidase alfa: enzyme therapy for Anderson–Fabry disease. Therapy 1:2, pages 203-211.
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