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Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study.
Wang, Bruce; Ventura, Paolo; Takase, Kei-Ichiro; Thapar, Manish; Cassiman, David; Kubisch, Ilja; Liu, Shangbin; Sweetser, Marianne T; Balwani, Manisha.
Afiliação
  • Wang B; Division of Gastroenterology, Department of Medicine, University of California San Francisco, San Francisco, CA, 94143, USA. Bruce.Wang@ucsf.edu.
  • Ventura P; University of Modena and Reggio Emilia, Modena, Italy.
  • Takase KI; Iizuka Hospital, Iizuka, Japan.
  • Thapar M; Thomas Jefferson University, Philadelphia, PA, USA.
  • Cassiman D; University Hospital Leuven, Leuven, Belgium.
  • Kubisch I; Klinikum Chemnitz, Chemnitz, Germany.
  • Liu S; Alnylam Pharmaceuticals, Cambridge, MA, USA.
  • Sweetser MT; Alnylam Pharmaceuticals, Cambridge, MA, USA.
  • Balwani M; Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Orphanet J Rare Dis ; 17(1): 327, 2022 08 26.
Article em En | MEDLINE | ID: mdl-36028858

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Porfirias Hepáticas / Porfiria Aguda Intermitente Idioma: En Ano de publicação: 2022 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Porfirias Hepáticas / Porfiria Aguda Intermitente Idioma: En Ano de publicação: 2022 Tipo de documento: Article