Raili Kauppinen, EAN 2020 – ENVISION Study in Patients With Acute Hepatic Porphyria
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).
Questions
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.
