EIGHTEEN-MONTH INTERIM ANALYSIS OF EFFICACY AND SAFETY OF GIVOSIRAN, AN RNAI THERAPEUTIC FOR ACUTE HEPATIC PORPHYRIA, IN THE ENVISION OPEN LABEL EXTENSION

EIGHTEEN-MONTH INTERIM ANALYSIS OF EFFICACY AND SAFETY OF GIVOSIRAN, AN RNAI THERAPEUTIC FOR ACUTE HEPATIC PORPHYRIA, IN THE ENVISION OPEN LABEL EXTENSION

Introduction: Acute hepatic porphyria (AHP) is a family of rare genetic diseases due to enzyme defects in hepatic heme biosynthesis. ENVISION is an ongoing phase 3 study, evaluating efficacy and safety of givosiran in symptomatic AHP patients. Objective: Describe efficacy and safety resuts of the EN...

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Bibliographic Details
Main Authors: D Kuter, S Keel, C Parker, DC Rees, U Stölzel, P Ventura, M Balwani, L Gouya, S Rhyee, S Silver
Format: Article
Language:English
Published: Elsevier 2021-10-01
Series:Hematology, Transfusion and Cell Therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2531137921001577

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http://www.sciencedirect.com/science/article/pii/S2531137921001577

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