Therapeutic Areas
Science of RNAi
Video
RNAi Mechanism of Action
This video highlights the mechanism of action (MOA) of RNAi therapeutics and its role in silencing gene expression. This video is intended to be viewed in its entirety.
Congresses
Transthyretin Amyloidosis (ATTR)
American Academy of Neurology (AAN) 2026
Cardiovascular (CV)
American College of Cardiology (ACC) 2026
Transthyretin Amyloidosis (ATTR)
American College of Cardiology (ACC) 2026
Publications
Transthyretin Amyloidosis (ATTR)
Effect of vutrisiran on components of health status in transthyretin amyloidosis with cardiomyopathy: the HELIOS-B study
European Journal of Heart Failure
Transthyretin Amyloidosis (ATTR)
Efficacy and Safety of Vutrisiran in Patients with Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy: Analysis of the East Asian Subpopulation from HELIOS-A
Neurology and Therapy
Primary Hyperoxaluria Type 1 (PH1)
Final Results of the ILLUMINATE-A Phase 3 Clinical Trial of Lumasiran for Primary Hyperoxaluria 1
Clinical Journal of the American Society of Nephrology
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Clinical Trials
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