AG10 CONSISTENTLY STABILIZES TRANSTHYRETIN TO A HIGH LEVEL IN BOTH WILD TYPE AND MUTANT AMYLOID CARDIOMYOPATHY: RESPONDER ANALYSES FROM A PHASE 2 CLINICAL TRIAL

2019 
AG10 is a highly selective and potent oral transthyretin (TTR) stabilizer under development for TTR amyloidosis (ATTR). ATTR cardiomyopathy (ATTR-CM) is under-diagnosed with no FDA-approved therapies indicated for its treatment. Hypothesis: At doses investigated, AG10 achieves complete stabilization
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