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:

  OBJECTIVE:
  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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