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Enzyme replacement therapy
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11	Microsoft PowerPoint - Koning CORDMarch22009Final.ppt [Compatibility Mode] Add to Reading List Source URL: www.raredisorders.ca Language: English - Date: 2009-04-08 12:51:18 Fabry disease Medicine Enzyme replacement therapy Lysosomal storage disease Fabry Robert J. Desnick Health Rare diseases Lipid storage disorders
12	Amicus Therapeutics Announces Second Quarter 2007 Financial Results Add to Reading List Source URL: www.fabry.org Language: English - Date: 2014-12-02 16:03:24 Rare diseases Fabry disease Lysosomal storage diseases Renal function Biomarker Enzyme replacement therapy Clinical trial ERT Glycogen storage disease type II Medicine Health Clinical research
13	Newborn Screening for Pompe Disease: Summary of the Condition Review Workgroup Report Alex R. Kemper, MD, MPH, MS May 17, 2013 Add to Reading List Source URL: www.hrsa.gov Language: English - Date: 2014-12-04 01:12:49 Glycogen storage disease type II Newborn screening Screening Enzyme replacement therapy Fabry disease Health Rare diseases Medicine
14	Review - Laronidase (Aldurazyme) Add to Reading List Source URL: www.fda.gov Language: English Mucopolysaccharidosis Hurler syndrome Iduronidase Lysosomal storage disease Coarse facial features BioMarin Pharmaceutical Enzyme replacement therapy Hurler–Scheie syndrome Methadone Health Syndromes Medicine
15	Microsoft PowerPoint - Fabry Figures reworked bb.ppt Add to Reading List Source URL: www.fabry.org Language: English - Date: 2014-12-04 03:16:52 Organ failure Lipid storage disorders Rare diseases Fabry disease Kidney diseases Chronic kidney disease Enzyme replacement therapy Renal function Lysosomal storage disease Health Anatomy Medicine
16	Microsoft Word - Document1 Add to Reading List Source URL: www.hrsa.gov Language: English - Date: 2014-12-04 01:13:21 Pediatrics Lipid storage disorders Medical genetics Lysosomal storage disease Newborn screening Inborn error of metabolism Genetic disorder Cystic fibrosis Enzyme replacement therapy Health Medicine Rare diseases
17	Journal List > NIHPA Author Manuscripts Mol Genet Metab. Author manuscript; available in PMC 2010 December 1. Published in final edited form as: Mol Genet Metab[removed]December; 101(4): 324–331. Published online 2010 Au Add to Reading List Source URL: amdapompe.ehclients.com Language: English - Date: 2011-05-07 19:52:59 Rare diseases Lysosomal storage diseases Lipid storage disorders Inborn errors of carbohydrate metabolism Glycogen storage disease type II Hepatology Autophagy Acid alpha-glucosidase Enzyme replacement therapy Biology Health Anatomy
18	Editorial For reprint orders, please contact [removed] Immune modulation in Pompe disease treated with enzyme replacement therapy Expert Rev. Clin. Immunol. 8(6), 497–[removed]) Add to Reading List Source URL: amdapompe.ehclients.com Language: English - Date: 2013-05-26 18:40:15 Anatomy Immune system Glycoproteins Glycogen storage disease type II Hepatology Rituximab Antibody Lysosomal storage disease Intravenous immunoglobulin Medicine Biology Immunology
19	Use of cardiac magnetic resonance imaging to evaluate cardiac structure, function and fibrosis in children with infantile Pompe disease on enzyme replacement therapy Add to Reading List Source URL: amdapompe.ehclients.com Language: English - Date: 2011-02-13 00:22:26 Cardiac imaging Rare diseases Cardiomyopathy Aging-associated diseases Medical emergencies Cardiac magnetic resonance imaging Glycogen storage disease type II Echocardiography Lysosomal storage disease Health Medicine Cardiology
20	The FASEB Journal • Research Communication Adjunctive albuterol enhances the response to enzyme replacement therapy in late-onset Pompe disease Dwight D. Koeberl,,1 Stephanie Austin, Laura E. Case,† Edward C. Smit Add to Reading List Source URL: amdapompe.ehclients.com Language: English - Date: 2014-08-13 23:54:32 Rare diseases Glycogen storage disease type II Hepatology Lysosomal storage disease Acid alpha-glucosidase Salbutamol Enzyme replacement therapy Methadone Medicine Chemistry Health