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Lysosomal storage disease
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31	The LINK Canadian Organization for Add to Reading List Source URL: www.raredisorders.ca Language: English - Date: 2009-04-01 16:11:41 Canadian Organization for Rare Disorders Lipid storage disorders European Organization for Rare Diseases Fabry disease Orphan drug National Organization for Rare Disorders Lysosomal storage disease Orphan Drug Act Medical genetics Medicine Health Rare diseases
32	PRESS RELEASE Contact: Brad Crittenden President, Canadian Association of Pompe Email: [removed] For Immediate Release Add to Reading List Source URL: www.raredisorders.ca Language: English Lysosomal storage diseases Glycogen storage disease type II Hepatology Durhane Wong-Rieger Orphan drug Orphan Drug Act Neglected diseases Rieger Health Medicine Rare diseases
33	EUROPEAN COMMISSION MEMO Brussels, 28 February[removed]Q&A on 26 new EU research projects on rare diseases Add to Reading List Source URL: europa.eu Language: English Medical terms National Institutes of Health Medical research Epidemiology Rare disease Orphan drug Gene therapy Lysosomal storage disease Chronic Medicine Health Biology
34	A Phase III Multicenter Study of SBC-‐102 (sebelipase alfa) in patients with Lysosomal Acid Lipase (LAL) Deficiency. Lysosomal Acid Add to Reading List Source URL: www.raredisorders.ca Language: English - Date: 2013-04-09 16:16:18 Medicine Cholesteryl ester storage disease Lysosomal Acid Lipase Deficiency Lipase Cirrhosis Hyperlipidemia Wolman Rare diseases Health Lipid storage disorders
35	The LINK Canadian Organization for Add to Reading List Source URL: www.raredisorders.ca Language: English - Date: 2009-04-01 16:11:55 Canadian Organization for Rare Disorders Morquio syndrome Orphan drug Lysosomal storage disease European Organization for Rare Diseases Fabry disease Rare Disease Day Glycogen storage disease type II Mental disorder Health Medicine Rare diseases
36	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
37	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
38	NIH OBA-RAC Update and Discussion of Human Gene Transfer Protocol #[removed]Administration of a Replication-Deficient AdenoAssociated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children wit Add to Reading List Source URL: osp.od.nih.gov Language: English - Date: 2013-12-16 10:09:50 Medicine Neuronal ceroid lipofuscinosis Lysosomal storage disease Molecular biology Biology Infantile neuronal ceroid lipofuscinosis Epilepsy Gene expression Lipid storage disorders Health Rare diseases
39	Nelson et al. Acta Neuropathologica Communications 2014, 2:20 http://www.actaneurocomms.org/content[removed]RESEARCH Open Access Add to Reading List Source URL: www.ncbi.nlm.nih.gov Language: English Cell biology Autophagy Programmed cell death Alpha-synuclein Fabry disease Neurodegeneration Lysosomal storage disease Biology Health Neurological disorders
40	EVIDENCE REVIEW: POMPE DISEASE Prepared for: THE ADVISORY COMMITTEE ON HERITABLE DISORDERS IN NEWBORNS AND CHILDREN FINAL V[removed] Add to Reading List Source URL: www.hrsa.gov Language: English - Date: 2014-12-04 01:12:52 Glycogen storage disease type II Lysosomal storage disease Acid alpha-glucosidase Newborn screening Screening Medical genetics Medicine Health Rare diseases