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)
Paula Del Río
Rosana Lucía Rey
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 .. The access to the whole abstract and if available the presentation file is available to FIP members and to congress participants of that specific congress.