In a small open-label observational study of 15 patients with ATTR cardiac amyloidosis (8 familial and 7 wild-type), 12 months of therapy with inotersen was associated with improved cardiac structure and function, improved submaximal exercise capacity, and decreased levels of brain natriuretic peptide biomarker when compared with baseline [37]. The gene discussed is NPPB; the disease is cardiac amyloidosis.