Efficacy and safety of givosiran in Japanese patients with acute hepatic porphyria: clinical findings from an expanded access study

Efficacy and safety of givosiran in Japanese patients with acute hepatic porphyria: clinical findings from an expanded access study

Abstract Acute hepatic porphyria (AHP), a rare genetic disorder, causes life-threatening porphyria attacks and chronic pain and impairs daily functioning and quality of life. Recently, a new siRNA therapy, givosiran, became available for AHP. This open-label, multicenter, single-arm study expanded a...

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Bibliographic Details
Main Authors: Nobuaki Ozaki, Yoshie Goto, Norihisa Fujii, Takuro Oyama, Karen Frascello, Ana Camejo, Tomohide Adachi
Format: Article
Language:English
Published: Nature Portfolio 2025-05-01
Series:Scientific Reports
Subjects:
Givosiran
Acute hepatic porphyria
Quality of life
Online Access:https://doi.org/10.1038/s41598-025-99526-3
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https://doi.org/10.1038/s41598-025-99526-3

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