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Expert Opinion on Biological Therapy Volume 2, 2002 - Issue 3
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Advances in the management of Anderson–Fabry disease:enzyme replacement therapy

Ravi Thadhani New York University School of Medicine, Neurogenetics Unit, 403 East 34th Street, New York, NY 10016, USA, Tel.: +1 212 263 8344; Fax: +1 212 263 8310;, E-mail: [email protected]
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Gregory M Pastores New York University School of Medicine, Neurogenetics Unit, 403 East 34th Street, New York, NY 10016, USA, Tel.: +1 212 263 8344; Fax: +1 212 263 8310;, E-mail: [email protected]
Pages 325-333 | Published online: 23 Feb 2005

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David J Clinkard, Hosam Amoodi, Vincent Y W Lin, Joseph M Chen & Julian M Nedzelski. (2011) Improved hearing with cochlear implantation in Fabry's disease. Cochlear Implants International 12:4, pages 238-240.
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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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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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Articles from other publishers (23)

Ley Nadine Lacbawan, Wei Zheng & Ozlem Goker‐Alpan. 2013. Enzyme Technologies. Enzyme Technologies 321 334 .
Melanie D. Sivley. (2013) Fabry Disease. Optometry and Vision Science 90:2, pages e63-e78.
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Gregory M. Pastores. 2012. Swaiman's Pediatric Neurology. Swaiman's Pediatric Neurology 403 451 .
Markus Cybulla, Kerstin Nanette Walter, Andreas Schwarting, Raffaelle DiVito, Sandro Feriozzi & Gere Sunder-Plassmann. (2009) Kidney transplantation in patients with Fabry disease. Transplant International 22:4, pages 475-481.
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Jayson D Edwards, Kraig S Bower, Dain B Brooks & Angela Walter. (2009) Fabry Disease and Chemosis. Cornea 28:2, pages 224-227.
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Thomas Thomaidis, Manfred Relle, Mitra Golbas, Christoph Brochhausen, Peter R. Galle, Michael Beck & Andreas Schwarting. (2009) Downregulation of ?-galactosidase A upregulates CD77: functional impact for Fabry nephropathy. Kidney International 75:4, pages 399-407.
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T. Kovacevic-Preradovic, M. Zuber, C.H. A. Jost, U. Widmer, B. Seifert, G. Schulthess, A. Fischer & R. Jenni. (2008) Anderson-Fabry disease: long-term echocardiographic follow-up under enzyme replacement therapy. European Journal of Echocardiography 9:6, pages 729-735.
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Hartmut Michels & Eugen Mengel. (2008) Lysosomal storage diseases as differential diagnoses to rheumatic disorders. Current Opinion in Rheumatology 20:1, pages 76-81.
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D. Hajioff, S. Hegemannn, G. Conti, M. Beck, G. Sunder-Plassmann, U. Widmer, A. Mehta & A. Keilmann. (2006) Agalsidase alpha and hearing in Fabry disease: data from the Fabry Outcome Survey. European Journal of Clinical Investigation 36:9, pages 663-667.
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Christine M Eng, Dominique P Germain, Maryam Banikazemi, David G Warnock, Christoph Wanner, Robert J Hopkin, Jan Bultas, Philip Lee, Katherine Sims, Scott E Brodie, Gregory M Pastores, Joerg M Strotmann & William R Wilcox. (2006) Fabry disease: Guidelines for the evaluation and management of multi-organ system involvement. Genetics in Medicine 8:9, pages 539-548.
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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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Joe T. R. Clarke. 2009. A Clinical Guide to Inherited Metabolic Diseases. A Clinical Guide to Inherited Metabolic Diseases 297 323 .
M. Beck, R. Ricci, U. Widmer, F. Dehout, A. Garcia de Lorenzo, C. Kampmann, A. Linhart, G. Sunder-Plassmann, G. Houge, U. Ramaswami, A. Gal & A. Mehta. (2004) Fabry disease: overall effects of agalsidase alfa treatment. European Journal of Clinical Investigation 34:12, pages 838-844.
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Roscoe O Brady & Raphael Schiffmann. (2004) Enzyme-replacement therapy for metabolic storage disorders. The Lancet Neurology 3:12, pages 752-756.
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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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Bj??rn Hoffmann, Dietrich Reinhardt & Berthold Koletzko. (2004) Effect of enzyme-replacement therapy on gastrointestinal symptoms in Fabry disease. European Journal of Gastroenterology & Hepatology 16:10, pages 1067-1069.
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F. Dehout, D. Roland, S. Treille de Granseigne, B. Guillaume & L. Van Maldergem. (2004) Relief of gastrointestinal symptoms under enzyme replacement therapy in patients with Fabry disease. Journal of Inherited Metabolic Disease 27:4, pages 499-505.
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A. Mehta, R. Ricci, U. Widmer, F. Dehout, A. Garcia de Lorenzo, C. Kampmann, A. Linhart, G. Sunder-Plassmann, M. Ries & M. Beck. (2004) Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey. European Journal of Clinical Investigation 34:3, pages 236-242.
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Robin J Ziegler, Scott M Lonning, Donna Armentano, Chester Li, David W Souza, Maribeth Cherry, Christine Ford, Christine M Barbon, Robert J Desnick, Guangping Gao, James M Wilson, Richard Peluso, Simon Godwin, Barrie J Carter, Richard J Gregory, Samuel C Wadsworth & Seng H Cheng. (2004) AAV2 Vector Harboring a Liver-Restricted Promoter Facilitates Sustained Expression of Therapeutic Levels of ?-Galactosidase A and the Induction of Immune Tolerance in Fabry Mice. Molecular Therapy 9:2, pages 231-240.
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D Hajioff, S Goodwin, R Quiney, J Zuckerman, KD MacDermot & A Mehta. (2007) Hearing improvement in patients with Fabry disease treated with agalsidase alfa. Acta Paediatrica 92, pages 28-30.
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J.Jeffrey Malatack, Deborah M Consolini & Eliel Bayever. (2003) The status of hematopoietic stem cell transplantation in lysosomal storage disease. Pediatric Neurology 29:5, pages 391-403.
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M. Jeyakumar, T. D. Butters, R. A. Dwek & F. M. Platt. (2002) Glycosphingolipid lysosomal storage diseases: therapy and pathogenesis. Neuropathology and Applied Neurobiology 28:5, pages 343-357.
Crossref
Mary Branton, Raphael Schiffmann & Jeffrey B. Kopp. (2002) Natural History and Treatment of Renal Involvement in Fabry Disease. Journal of the American Society of Nephrology 13:suppl 2, pages S139-S143.
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