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Enzyme replacement therapy
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1	Migalastat and Enzyme Replacement Therapy Have Comparable Effects on Renal Function in Fabry Disease: Phase 3 Study Results Nicholls, K1*, Bichet, DG2, Giugliani, R3, Hughes, D4, Schiffmann, R5, Wilcox, W6, Skuban, N7, R Add to Reading List Source URL: files.shareholder.com
2	The Journal of Rare Disorders CochraneCorner Add to Reading List Source URL: www.journalofraredisorders.com Language: English - Date: 2015-01-07 11:10:15 Lipid storage disorders Fabry disease Alpha-galactosidase Enzyme replacement therapy Globotriaosylceramide Placebo Health Medicine Rare diseases
3	7 November 2014 Dear Committee Re: Life Saving Drugs Programme Review Fabry Support Group of Australia Submission Add to Reading List Source URL: pbs.gov.au Language: English - Date: 2015-04-10 00:39:25 Medicine Clinical research Fabry disease Enzyme replacement therapy Methadone Clinical trial ERT Health Rare diseases Lipid storage disorders
4	Minimum Recommended Schedule of Assessments for MPS I Patients Initial Assessments Every 6 Add to Reading List Source URL: www.lsdregistry.net Language: English - Date: 2013-04-11 15:29:33 Hematology Hematopoietic stem cell transplantation Lymphatic system Surgical oncology Enzyme replacement therapy Graft-versus-host disease Genzyme Medicine Health Transplantation medicine
5	Pancreatic enzyme replacement therapy Add to Reading List Source URL: www.health.qld.gov.au Language: English - Date: 2014-05-07 02:17:19 Pancreas disorders Applied sciences Food science Chronic pancreatitis Nutrition Dietitian Enzyme Pancreatectomy Medicine Health Health sciences
6	7 November 2014 Dear Committee Re: Life Saving Drugs Programme Review Fabry Support Group of Australia Submission Add to Reading List Source URL: www.pbs.gov.au Language: English - Date: 2015-03-04 00:09:47 Medicine Clinical research Fabry disease Enzyme replacement therapy Methadone Clinical trial ERT Health Rare diseases Lipid storage disorders
7	Enzyme replacement therapy for Gaucher’s disease The clinical effectiveness and cost-effectiveness of enzyme replacement therapy for Gaucher’s disease: a systematic review Add to Reading List Source URL: www.journalslibrary.nihr.ac.uk Language: English - Date: 2013-05-21 08:23:39 Rare diseases Lipid storage disorders Clinical research Healthcare National Institute for Health and Clinical Excellence Quality of life National Health Service Health technology assessment Quality-adjusted life year Health Medicine Health economics
8	Health Technology Assessment 2006; Vol. 10: No. 24 Enzyme replacement therapy for Gaucher’s disease Feedback The HTA Programme and the authors would like to know your views about this report. Add to Reading List Source URL: www.journalslibrary.nihr.ac.uk Language: English - Date: 2013-05-21 08:23:19 Health technology assessment National Institute for Health and Clinical Excellence Quality of life National Health Service Quality-adjusted life year Cost-effectiveness analysis SF-36 Centre for Reviews and Dissemination Cost–utility analysis Health Health economics Medicine
9	SAGE-Hindawi Access to Research Stroke Research and Treatment Volume 2011, Article ID[removed], 7 pages doi:[removed][removed]Review Article Add to Reading List Source URL: downloads.hindawi.com Language: English - Date: 2014-03-26 10:08:06 Rare diseases Fabry disease Enzyme replacement therapy Lysosomal storage disease Stroke Aspirin Fabry Robert J. Desnick Health Medicine Lipid storage disorders
10	Technology Offer Ref.-No.0212 Use of transglutaminase 1-liposomes for topical enzyme replacement therapy for transglutaminase 1 deficient lamellar ichthyosis Add to Reading List Source URL: www.fmp-berlin.info Language: English - Date: 2012-11-09 08:31:39 Rare diseases Autoantigens Medicine Transglutaminase Lamellar ichthyosis Ichthyosis Liposome Keratin Lamella Genodermatoses Biology Health