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We are joined by Prof Marianna Fontana (University College London, UK), as she outlines the key findings from the phase 3 APOLLO-B study (Alnylam Pharmaceuticals).

APOLLO-B investigated the use of patisiran, a transthyretin-directed small interfering RNA, in a cohort of patients with cardiac amyloidosis. 360 patients were enrolled in the trial and were randomized 1:1 to receive either intravenous patisiran or a placebo.

Interview Questions:
1. What is the importance of this study?
2. What is the mechanism of action of the study drug (patisiran)?
4. What was study design, outcome measures and patient population?
5. What are the findings to date? What conclusions can be made?
6. Are there any specific patient subgroups with cardiomyopathy for whom patisiran may be more or less effective?
7. What are the next steps?

Recorded on-site at HFA 2023, Prague.

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