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Breaking the chains of lipoprotein lipase deficiency: A pediatric perspective on the efficacy and safety of Volanesorsen.
den Hollander, Bibiche; Brands, Marion M; Nijhuis, Ilse J M; Doude van Troostwijk, Lous J A E; van Essen, Peter; Hofsteenge, Geesje H; Koot, Bart G; Müller, Annelieke R; Tseng, Laura A; Stroes, Erik S G; van de Ven, Peter M; Wiegman, Albert; van Karnebeek, Clara D M.
  • den Hollander B; Amsterdam UMC location University of Amsterdam, Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Meibergdreef 9, Amsterdam, the Netherlands; Amsterdam UMC, Emma Center for Personalized Medicine, Amsterdam, the Netherlands; United for Metabolic
  • Brands MM; Amsterdam UMC location University of Amsterdam, Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Meibergdreef 9, Amsterdam, the Netherlands; Amsterdam UMC, Emma Center for Personalized Medicine, Amsterdam, the Netherlands; United for Metabolic
  • Nijhuis IJM; Wilhelmina Hospital Assen, Department of Pediatrics, Europaweg-Zuid 1, Assen, the Netherlands.
  • Doude van Troostwijk LJAE; Wilhelmina Hospital Assen, Department of Pharmacy, Europaweg-Zuid 1, Assen, the Netherlands.
  • van Essen P; Radboud University Medical Center, Department of Pediatrics, Amalia Children's Hospital, Geert Grooteplein Zuid 10, Nijmegen, the Netherlands.
  • Hofsteenge GH; Amsterdam UMC location University of Amsterdam, Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Meibergdreef 9, Amsterdam, the Netherlands; Amsterdam UMC location University of Amsterdam, Department of Nutrition & Dietetics, Meibergdreef 9
  • Koot BG; Amsterdam UMC location University of Amsterdam, Department of Paediatric Gastroenterology and Nutrition, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Meibergdreef 9, Amsterdam, the Netherlands.
  • Müller AR; Amsterdam UMC location University of Amsterdam, Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Meibergdreef 9, Amsterdam, the Netherlands; Amsterdam UMC, Emma Center for Personalized Medicine, Amsterdam, the Netherlands.
  • Tseng LA; Amsterdam UMC location University of Amsterdam, Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Meibergdreef 9, Amsterdam, the Netherlands; Amsterdam UMC, Emma Center for Personalized Medicine, Amsterdam, the Netherlands; University Medical Ce
  • Stroes ESG; Amsterdam UMC location University of Amsterdam, Department of Vascular Medicine, Meibergdraaf 9, Amsterdam, the Netherlands.
  • van de Ven PM; University Medical Centre Utrecht, Department of Data Science and Biostatistics, Julius Center for Health Sciences and Primary Care, Heidelberglaan 100, Utrecht, Netherlands.
  • Wiegman A; Amsterdam UMC location University of Amsterdam, Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Meibergdreef 9, Amsterdam, the Netherlands.
  • van Karnebeek CDM; Amsterdam UMC location University of Amsterdam, Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Meibergdreef 9, Amsterdam, the Netherlands; Amsterdam UMC, Emma Center for Personalized Medicine, Amsterdam, the Netherlands; United for Metabolic
Mol Genet Metab ; 142(1): 108347, 2024 May.
Article en En | MEDLINE | ID: mdl-38401382

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Oligonucleótidos / Pancreatitis / Triglicéridos / Hiperlipoproteinemia Tipo I Límite: Adolescent / Female / Humans Idioma: En Año: 2024 Tipo del documento: Article

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Oligonucleótidos / Pancreatitis / Triglicéridos / Hiperlipoproteinemia Tipo I Límite: Adolescent / Female / Humans Idioma: En Año: 2024 Tipo del documento: Article