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Sebelipase alfa for lysosomal acid lipase deficiency: 5-year treatment experience from a phase 2 open-label extension study.
Malinová, Vera; Balwani, Manisha; Sharma, Reena; Arnoux, Jean-Baptiste; Kane, John; Whitley, Chester B; Marulkar, Sachin; Abel, Florian.
Affiliation
  • Malinová V; Department for Metabolic Diseases, Children's Clinic, General Faculty Hospital and First Faculty of Medicine of Charles University in Prague, Prague, Czech Republic.
  • Balwani M; Department of Genetics and Genomic Sciences and Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • Sharma R; Department of Endocrinology and Metabolic Medicine, Salford Royal Foundation NHS Trust, Salford, UK.
  • Arnoux JB; Department of Inherited Metabolic Diseases, Hôpital Necker-Enfants Malades, Paris, France.
  • Kane J; Department of Medicine, University of California San Francisco, San Francisco, CA, USA.
  • Whitley CB; Advanced Therapies Program, and Gene Therapy Center, University of Minnesota, Minneapolis, MN, USA.
  • Marulkar S; Alexion Pharmaceuticals, Inc., Boston, MA, USA.
  • Abel F; Alexion Pharmaceuticals, Inc., Boston, MA, USA.
Liver Int ; 40(9): 2203-2214, 2020 09.
Article in En | MEDLINE | ID: mdl-32657505
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Wolman Disease / Sterol Esterase Limits: Adult / Humans Language: En Journal: Liver Int Journal subject: GASTROENTEROLOGIA Year: 2020 Type: Article Affiliation country: Czech Republic
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Wolman Disease / Sterol Esterase Limits: Adult / Humans Language: En Journal: Liver Int Journal subject: GASTROENTEROLOGIA Year: 2020 Type: Article Affiliation country: Czech Republic