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Lipid therapy
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#	Item
31	Carnitine Supplementation and VLCAD Deficiency There has been considerable controversy regarding the role of Lcarnitine (Carnitor®, Sigma-Tau Pharmaceuticals, Inc.) and its value as a supplemental therapy in fat oxidati Add to Reading List Source URL: www.fodsupport.org Language: English - Date: 2012-05-31 13:41:07 Dietary supplements Quaternary ammonium compounds Medium-chain acyl-coenzyme A dehydrogenase deficiency Carnitine Very long-chain acyl-coenzyme A dehydrogenase deficiency Rare diseases Fatty-acid metabolism disorder Inborn error of lipid metabolism Health Chemistry Medicine
32	http://pqasb.pqarchiver.com/latimes/access[removed]html?dids=3 Add to Reading List Source URL: www.canavan.org Language: English - Date: 2009-11-06 14:09:34 Medical genetics Rare diseases Lipid storage disorders Canavan disease Leukodystrophies Emerging technologies Gene therapy N-Acetylaspartic acid Genetic disorder Biology Health Medicine
33	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
34	NNPDF-Funded Research Grant # 40 TITLE: Lysosomal Storage Disease Research Consortium Contribution for NNPDF to Join Consortium PROJECT INVESTIGATOR: Multiple - See "More Details" PERIOD: [removed]2006 PROJECT DES Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2010-04-26 15:39:01 Rare diseases Biotechnology Applied genetics Medical genetics Lysosomal storage disease Gangliosidosis GM2 gangliosidoses Substrate reduction therapy Ganglioside Biology Health Lipid storage disorders
35	Grant #45 Frances M. Platt University of Oxford Evaluation of anti-inflammatory intervention (NSAIDS) as an adjunctive therapy in Niemann-Pick type C1 disease Lysosomal storage diseases are severe disorders that typicall Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2010-04-26 15:39:37 Medicine Neurodegenerative disorders Non-steroidal anti-inflammatory drugs Lysosomal storage disease Niemann–Pick disease NPC1 Miglustat Sandhoff disease Aspirin Health Lipid storage disorders Rare diseases
36	NNPDF-Funded Research Grant # 32 TITLE: Therapeutic Gene Delivery to the Cerebellum in Niemann-Pick Type C PROJECT INVESTIGATOR: Timothy M. Cox, MD, FRCP, FMedSci & Robin Henry Lachmann, M.A., M.B., Bchir, Ph.D. PERIOD: Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2010-04-26 15:37:58 Medicine NPC1 Lipid storage disorders Rare diseases Medical terms Niemann–Pick disease Purkinje cell Neuron Gene therapy Biology Anatomy Cerebellum
37	SYNAGEVA’S SEBELIPASE ALFA RECEIVES BREAKTHROUGH THERAPY DESIGNATION FOR EARLY ONSET LAL DEFICIENCY FROM THE U.S. FOOD AND DRUG ADMINISTRATION LEXINGTON, Mass., May 20, [removed]Synageva BioPharma Corp. (Synageva) (NASD Add to Reading List Source URL: www.synageva.com Language: English - Date: 2013-05-20 07:13:10 Wolman disease Lysosomal Acid Lipase Deficiency Cholesteryl ester storage disease Lysosomal storage disease Mucopolysaccharidosis Sanfilippo syndrome Wolman Acid lipase disease Lysosomal lipase Health Rare diseases Lipid storage disorders
38	SYNAGEVA BIOPHARMA™ HIGHLIGHTS DATA AT THE NASPGHAN MEETING LEXINGTON, Mass., October 11, [removed]Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a biopharmaceutical company developing therapeutic products for rare Add to Reading List Source URL: www.synageva.com Language: English - Date: 2013-10-11 07:24:04 Lipid storage disorders Lysosomal Acid Lipase Deficiency Wolman disease Cholesteryl ester storage disease Sanfilippo syndrome Lysosomal storage disease Hunter syndrome Enzyme replacement therapy Mucopolysaccharidosis Health Medicine Rare diseases
39	Step 7 Monitoring statin therapy. A high level of randomized controlled trial (RCT) evidence supports the use of an initial fasting lipid panel (total cholesterol, triglycerides, HDL–C, and calculated LDL–C), foll Add to Reading List Source URL: www.ksw-gtg.com Language: English - Date: 2014-07-24 09:34:59 Medicine Cardiology Carboxylic acids Diols Lactones Hypercholesterolemia Low-density lipoprotein Simvastatin Rosuvastatin Chemistry Organic chemistry Statins
40	Department of Human Genetics Division of Medical Genetics www.genetics.emory.edu Fabry Disease: Important Facts for Women Add to Reading List Source URL: genetics.emory.edu Language: English - Date: 2009-03-30 13:10:53 Rare diseases Fabry disease Lysosomal storage disease Enzyme replacement therapy Medical genetics Genetic disorder Alpha-galactosidase Fabry Genzyme Medicine Health Lipid storage disorders

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