Raili Kauppinen (University Hospital of Helsinki, Finland) shares key findings and insights into the ENVISION study, which evaluated the efficacy and safety of givosiran, a subcutaneously administered RNA interference therapeutic targeting aminolevulinic acid synthase 1, in patients with acute hepatic porphyrias (ClinicalTrials.gov Identifier: NCT03338816).
1. Could you give us a brief overview of acute hepatic porphyria, its pathophysiology and manifestations? (0:05)
2. What is givosiran and what is its mechanism of action? (2:12)
3. What were the aims and design of the ENVISION study? (2:50)
4. What have been the main efficacy and safety findings of this study? (4:03)
5. What has been the impact of this on patients’ lives and on the current treatment paradigm? (5:01)
Speaker disclosure: Raili Kauppinen reports receiving advisory board fees from Alnylam Pharmaceuticals, advisory fees from Recordati Rare Diseases, travel fees from Sanofi, and ownership of stock in the Orion Corporation.
Support: Interview and filming supported by Touch Medical Media.
Filmed at the 6th Congress of the European Academy of Neurology (EAN) and 1st EAN Virtual Congress, May 2020.
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