Sebelipase alfa over 52 weeks reduces serum transaminases,liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency |
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| Authors: | Vassili Valayannopoulos,Vera Malinova,Tomas Honzí k,Manisha Balwani,Catherine Breen,Patrick B. Deegan,Gregory M. Enns,Simon A. Jones,John P. Kane,Eveline O. Stock,Radhika Tripuraneni,Stephen Eckert,Eugene Schneider,Gavin Hamilton,Michael S. Middleton,Claude Sirlin,Bruce Kessler,Christopher Bourdon,Simeon A. Boyadjiev,Reena Sharma,Chris Twelves,Chester B. Whitley,Anthony G. Quinn |
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| Affiliation: | 1 Ref Centre IEM, Necker-Enf Malades Hosp, IMAGINE Institute and Paris Descartes University, Paris, France;2 Department of Pediatrics, 1st Faculty of Medicine, Charles University and General University Hospital, Prague, Czech Republic;3 Department of Genetics and Genomic Sciences, The Mount Sinai School of Medicine, New York, NY, United States;4 Manchester Centre for Genomic Medicine, St. Mary’s Hospital, Manchester, UK;5 Department of Medicine, Addenbrooke’s Hospital, Cambridge, UK;6 Department of Pediatrics, Lucile Packard Children’s Hospital, Stanford University, Stanford, CA, United States;7 Divisions of Endocrinology & Metabolism, University of California, San Francisco, CA, United States;8 Divisions of Cardiology, University of California, San Francisco, CA, United States;9 Synageva BioPharma Corp., Lexington, MA, United States;10 University of California, San Diego, CA, United States;11 Eureka Internal Medicine, Eureka, CA, United States;12 Health Sciences North, Sudbury, Ontario, Canada;13 University of California, Davis Medical Center, Department of Pediatrics, Section of Genetics, Davis, CA, United States;14 Salford Royal NHS Foundation Trust, Salford, UK;15 Leeds Institute of Cancer and Pathology, Leeds, UK;16 University of Minnesota, Minneapolis, MN, United States |
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| Abstract: | |
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| Keywords: | LAL, lysosomal acid lipase LAL D, lysosomal acid lipase deficiency CE, cholesteryl ester TG, triglycerides NAFLD, non-alcoholic fatty liver disease NASH, non-alcoholic steatohepatitis MRI, magnetic resonance imaging MRS, magnetic resonance spectroscopy ALT, alanine aminotransferase AST, aspartate aminotransferase LDL, low-density lipoprotein HDL, high-density lipoprotein GGT, gamma glutamyl transferase PDFF, proton density fat fraction AE, adverse event CTCAE, common terminology criteria for adverse events IRR, infusion-related reaction MN, multiples of normal |
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