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Lysosomal storage disease
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121	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
122	SYNAGEVA BIOPHARMA™ HIGHLIGHTS DATA PRESENTATIONS AND SATELLITE SYMPOSIUM AT THE EUROPEAN ATHEROSCLEROSIS SOCIETY CONGRESS LEXINGTON, Mass., June 3, [removed]Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a biopharm Add to Reading List Source URL: www.synageva.com Language: English - Date: 2014-06-03 10:39:51 Medicine Lysosomal Acid Lipase Deficiency Wolman disease Cholesteryl ester storage disease Sanfilippo syndrome Mucopolysaccharidosis Lysosomal storage disease Atherosclerosis Low-density lipoprotein Health Rare diseases Lipid storage disorders
123	Differential Diagnosis of NAFLD- A Short Summary: Almost a fifth of our general pediatric population is now classified as overweight in the United States. When such children present with elevated liver enzymes the most i Add to Reading List Source URL: www.naspghan.org Language: English - Date: 2014-06-18 12:28:22 Hepatology Rare diseases Histopathology Hepatitis Cholesteryl ester storage disease Non-alcoholic fatty liver disease Fatty liver Lysosomal Acid Lipase Deficiency Cirrhosis Medicine Health Lipid storage disorders
124	Guide to Understanding Multiple Sulfatase Deficiency (MSD[removed]removed] www.mpssociety.org.uk Add to Reading List Source URL: www.mpssociety.org.uk Language: English - Date: 2013-06-10 10:14:46 Genetic genealogy Medical genetics Mucopolysaccharidosis Multiple sulfatase deficiency Lysosomal storage disease Sulfatase Syndromes Health Rare diseases Lipid storage disorders
125	Chair Rohit Kohli, MBBS, MS Associate Professor of Pediatrics Medical Director, Complex Surgery and Transplant Inpatient Unit Co-Director, Steatohepatitis Center Add to Reading List Source URL: www.naspghan.org Language: English - Date: 2014-06-18 09:22:16 Hepatology Lipid storage disorders Rare diseases Hepatitis Cholesteryl ester storage disease Non-alcoholic fatty liver disease Cirrhosis Lysosomal Acid Lipase Deficiency Wolman disease Medicine Health Anatomy
126	What’s New in Newborn Screening? Funded by: Illinois Department of Public Health Add to Reading List Source URL: www.idph.state.il.us Language: English - Date: 2014-04-16 11:46:52 Lipid storage disorders Pediatrics Syndromes Neurodegenerative disorders Lysosomal storage disease Glycogen storage disease type II Newborn screening Hunter syndrome Niemann–Pick disease Health Rare diseases Medicine
127	The people. Their lives. The impact[removed]Annual Report Dear Fellow Shareholders, 2013 was an amazing year for both Synageva and for patients. We met, Add to Reading List Source URL: www.synageva.com Language: English - Date: 2014-04-23 16:54:16 Lipid storage disorders Lysosomal Acid Lipase Deficiency Wolman disease Cholesteryl ester storage disease Orphan drug Hunter syndrome Clinical trial Cirrhosis Sanfilippo syndrome Health Rare diseases Medicine
128	Chair Rohit Kohli, MBBS, MS Associate Professor of Pediatrics Medical Director, Complex Surgery and Transplant Inpatient Unit Co-Director, Steatohepatitis Center Add to Reading List Source URL: www.naspghan.org Language: English - Date: 2014-06-27 11:01:50 Hepatology Lipid storage disorders Rare diseases Hepatitis Cholesteryl ester storage disease Non-alcoholic fatty liver disease Cirrhosis Lysosomal Acid Lipase Deficiency Wolman disease Medicine Health Anatomy
129	Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1ⴚ/ⴚ mouse Benny Liua, Stephen D. Turleya, Dennis K. Burnsb, Anna M. Millerc, Joyce J. Repaa,c, a Add to Reading List Source URL: www.parseghian.org Language: English - Date: 2009-08-07 14:59:34 NPC1 Chemistry Lipid storage disorders Rare diseases Steroids Sterols Cholesterol Niemann–Pick disease Low-density lipoprotein Biology Health Transcription factors
130	Pathways: Volume 6 No. 1 Summer 2004 A publication of ISMRD Add to Reading List Source URL: www.ismrd.org Language: English - Date: 2014-09-02 17:37:51 Medical genetics Lysosomal storage disease Glycoproteinosis Sialidosis Alpha-mannosidosis Fucosidosis Pseudo-Hurler polydystrophy Galactosialidosis I-cell disease Health Rare diseases Genetic genealogy

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