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- Act Early: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young. Andrew Kates, M.D. Sponsor: Eidos Therapeutics- CURRENTLY ENROLLING
- An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy). Dr. Joshua Mitchell Sponsor: Alnylam Pharmaceuticals- CURRENTLY ENROLLING