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Lysosomal storage disease
Results: 211

#	Item
51	Research Presented 2008 Lysosomal Diseases and the Brain Conference Organized by the Children’s Gaucher Research Fund --- Speaker Abstracts --Molecular Mechanisms of Lysosome Biogenesis Juan S. Bonifacino, Ph.D. - juan Add to Reading List Source URL: www.childrensgaucher.org Language: English - Date: 2010-07-24 10:47:39 Lipid storage disorders Rare diseases Cellular processes Lysosomal storage disease Lysosome Autophagy Endosome Mannose 6-phosphate Substrate reduction therapy Biology Cell biology Organelles
52	Gene Therapy for the CNS Manifestations of the Pediatric Lysosomal Storage Disorders Add to Reading List Source URL: osp.od.nih.gov Language: English - Date: 2014-01-08 14:46:06 Applied genetics Neuronal ceroid lipofuscinosis Gene delivery Lysosomal storage disease Medical research Gene therapy Biology Health Medicine
53	Attacking a rare disease at its source with gene therapy Add to Reading List Source URL: medicalxpress.com Language: English - Date: 2014-11-27 14:56:26 Emerging technologies Applied genetics Bioethics Medical genetics Molecular biology Mucopolysaccharidosis Gene therapy Lysosomal storage disease Neuroglia Biology Medicine Health
54	13084 MPS III v6:Layout 1 Add to Reading List Source URL: www.mpssociety.org Language: English - Date: 2011-09-13 15:27:28 Rare diseases Sanfilippo syndrome Mucopolysaccharidosis Lysosomal storage disease Infection Diarrhea Hunter syndrome Hurler syndrome Health Medicine Syndromes
55	van Gelder et al. BMC Musculoskeletal Disorders 2013, 14(Suppl 2):P19 http://www.biomedcentral.com[removed]S2/P19 POSTER PRESENTATION Open Access Add to Reading List Source URL: amdapompe.ehclients.com Language: English - Date: 2013-07-21 20:00:12 Medicine Glycogen storage disease type II Hepatology Health Rare diseases Lysosomal storage diseases
56	NIH Public Access Author Manuscript Am J Med Genet C Semin Med Genet. Author manuscript; available in PMC 2013 February 15. NIH-PA Author Manuscript Add to Reading List Source URL: amdapompe.ehclients.com Language: English - Date: 2014-03-02 20:04:03 Autophagy Programmed cell death Inborn errors of carbohydrate metabolism Rare diseases Hepatology Glycogen storage disease type II Acid alpha-glucosidase Lysosomal storage disease Lysosome Biology Cell biology Cellular processes
57	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
58	Molecular Bases of Disease: Glycosylation-independent Lysosomal Targeting of Acid α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice John A. Maga, Jianghong Zhou, Ravi Kambampati, Susan Peng, Xu Wang, Richar Add to Reading List Source URL: amdapompe.ehclients.com Language: English - Date: 2013-07-21 20:12:27 Peptide hormones Hepatology Glycogen storage disease type II Mannose 6-phosphate Insulin-like growth factor 1 Insulin-like growth factor BioMarin Pharmaceutical Lysosomal storage disease Alpha-glucosidase Biology Cell biology Growth factors
59	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
60	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