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Givosiran and hereditary coproporphyria: case report

  • At: Seville (Spain) (2022)
  • Type: Poster
  • Poster code: HPS-065
  • By: RAMIS, Marina Barbara (Marques de Valdecilla University Hospital, Spain)
  • Co-author(s): Marina Barbara Ramis (Marques de Valdecilla University Hospital, Santander, Spain)
    Víctor Fernández
    Lorenzo Pérez-Periáñez
    Irene Aragón
    Paula Del Río
    María Rioja
    Jesús Mayorga
    Marta Valero
    Clara Fernández
    Rosana Lucía Rey
  • Abstract:

    Givosiran is a therapeutic small interfering RNA (siRNA) that inhibits hepatic delta-aminolevulinic acid synthase 1 (ALAS1). In patients with acute hepatic porphyria, induction of hepatic ALAS1 results in the accumulation of neurotoxic heme intermediates, including delta-aminolevulinic acid and porphobilinogen.
    The aim of this study was ..

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Last update 28 September 2023

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