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Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency.
Valayannopoulos, Vassili; Malinova, Vera; Honzík, Tomas; Balwani, Manisha; Breen, Catherine; Deegan, Patrick B; Enns, Gregory M; Jones, Simon A; Kane, John P; Stock, Eveline O; Tripuraneni, Radhika; Eckert, Stephen; Schneider, Eugene; Hamilton, Gavin; Middleton, Michael S; Sirlin, Claude; Kessler, Bruce; Bourdon, Christopher; Boyadjiev, Simeon A; Sharma, Reena; Twelves, Chris; Whitley, Chester B; Quinn, Anthony G.
Afiliación
  • Valayannopoulos V; Ref Centre IEM, Necker-Enf Malades Hosp, IMAGINE Institute and Paris Descartes University, Paris, France.
  • Malinova V; Department of Pediatrics, 1st Faculty of Medicine, Charles University and General University Hospital, Prague, Czech Republic.
  • Honzík T; Department of Pediatrics, 1st Faculty of Medicine, Charles University and General University Hospital, Prague, Czech Republic.
  • Balwani M; Department of Genetics and Genomic Sciences, The Mount Sinai School of Medicine, New York, NY, United States.
  • Breen C; Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester, UK.
  • Deegan PB; Department of Medicine, Addenbrooke's Hospital, Cambridge, UK.
  • Enns GM; Department of Pediatrics, Lucile Packard Children's Hospital, Stanford University, Stanford, CA, United States.
  • Jones SA; Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester, UK.
  • Kane JP; Divisions of Endocrinology & Metabolism, University of California, San Francisco, CA, United States.
  • Stock EO; Divisions of Cardiology, University of California, San Francisco, CA, United States.
  • Tripuraneni R; Synageva BioPharma Corp., Lexington, MA, United States.
  • Eckert S; Synageva BioPharma Corp., Lexington, MA, United States.
  • Schneider E; Synageva BioPharma Corp., Lexington, MA, United States.
  • Hamilton G; University of California, San Diego, CA, United States.
  • Middleton MS; University of California, San Diego, CA, United States.
  • Sirlin C; University of California, San Diego, CA, United States.
  • Kessler B; Eureka Internal Medicine, Eureka, CA, United States.
  • Bourdon C; Health Sciences North, Sudbury, Ontario, Canada.
  • Boyadjiev SA; University of California, Davis Medical Center, Department of Pediatrics, Section of Genetics, Davis, CA, United States.
  • Sharma R; Salford Royal NHS Foundation Trust, Salford, UK.
  • Twelves C; Leeds Institute of Cancer and Pathology, Leeds, UK.
  • Whitley CB; University of Minnesota, Minneapolis, MN, United States.
  • Quinn AG; Synageva BioPharma Corp., Lexington, MA, United States. Electronic address: Anthony.Quinn@synageva.com.
J Hepatol ; 61(5): 1135-42, 2014 Nov.
Article en En | MEDLINE | ID: mdl-24993530
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Enfermedad de Wolman / Esterol Esterasa Tipo de estudio: Clinical_trials Límite: Adult / Female / Humans / Male / Middle aged Idioma: En Revista: J Hepatol Asunto de la revista: GASTROENTEROLOGIA Año: 2014 Tipo del documento: Article País de afiliación: Francia
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Enfermedad de Wolman / Esterol Esterasa Tipo de estudio: Clinical_trials Límite: Adult / Female / Humans / Male / Middle aged Idioma: En Revista: J Hepatol Asunto de la revista: GASTROENTEROLOGIA Año: 2014 Tipo del documento: Article País de afiliación: Francia