The primary end point was response rate to treatment (non-progression of neuropathy or cardiomyopathy), with responders defined as patients with mBMI reductions of <10% and NIS-LL changes of <2 points (in patients with TTR-FAP) or NT-proBNP concentration increases of <30% or <300 pg mL–1 (in patients with isolated cardiomyopathy). The gene discussed is TTR; the disease is cardiomyopathy.