Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study

Abstract Background Acute hepatic porphyria is a group of multisystem disorders of which acute intermittent porphyria is the most common subtype. Givosiran, a subcutaneously administered RNA interference therapeutic targeting liver ALAS mRNA, is approved for treating these disorders. This Phase 1/2...

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Bibliographic Details

Published in:	Orphanet Journal of Rare Diseases
Main Authors:	Eliane Sardh, Manisha Balwani, David C. Rees, Karl E. Anderson, Gang Jia, Marianne T. Sweetser, Bruce Wang
Format:	Article
Language:	English
Published:	BMC 2024-10-01
Subjects:	Acute hepatic porphyria (AHP) Acute intermittent porphyria (AIP) Givosiran RNA interference Delta-aminolevulinic acid (ALA) Porphobilinogen (PBG)
Online Access:	https://doi.org/10.1186/s13023-024-03284-w