Home > Raili Kauppinen, EAN 2020 – ENVISION Study in Patients With Acute Hepatic Porphyria
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Raili Kauppinen, EAN 2020 – ENVISION Study in Patients With Acute Hepatic Porphyria

Published Online: June 5th 2020

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.

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