Effectiveness and tolerability of givosiran for the management of acute hepatic porphyria: A monocenter real-life evaluation-Reference-Cited by-同舟云学术

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Effectiveness and tolerability of givosiran for the management of acute hepatic porphyria: A monocenter real-life evaluation

Published:2024-09 Issue: Volume:40 Page:101111
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Guida Claudio Carmine,Nardella Maria,del Mar YS Perez Aurora,Savino Maria,Ferrara Gaetano,Napolitano Francesco,Crisetti Annalisa,Aucella Francesco,Aucella Filippo

Funder

Alnylam Pharmaceuticals

Publisher

Elsevier BV

Reference23 articles.

1. Acute hepatic porphyrias: recommendations for evaluation and long-term management;Balwani;Hepatology,2017

2. Phase 3 trial of RNAi therapeutic givosiran for acute intermittent porphyria;Balwani;N. Engl. J. Med.,2020

3. Acute intermittent porphyria: an overview of therapy developments and future perspectives focusing on stabilisation of HMBS and proteostasis regulators;Bustad;Int. J. Mol. Sci.,2021

4. Laboratory diagnosis of porphyria;Di Pierro;Diagnostics (Basel).,2021

5. EXPLORE: a prospective, multinational, natural history study of patients with acute hepatic porphyria with recurrent attacks;Gouya;Hepatology,2020

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