Risk Factors for Mortality in Patients with Hereditary Transthyretin-Mediated Amyloidosis: An Analysis of APOLLO and Global Open Label Extension Studies

Hereditary transthyretin-mediated (hATTR) amyloidosis is a rapidly progressive, fatal disease caused by transthyretin gene mutations resulting in amyloid deposition in multiple organs and tissues. Heterogenous clinical manifestations occur and most patients (pts) develop a mixed phenotype of polyneuropathy and cardiomyopathy. Patisiran, an RNAi therapeutic, was investigated in APOLLO (NCT01960348), the largest study in pts with hATTR amyloidosis...