Article

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

2024-10-28

Abstract excerpt

<title>Abstract</title> <p>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 access to givosiran and further explored its safety and efficacy in 10 Japanese patients with AHP....

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Literature Corpus work: d3961503-958f-59f9-86d6-6a5d97cc8448
DOI: 10.21203/rs.3.rs-5201348/v1

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