Applying the response criteria by Ohlow et al., including the improvement of ≥2 parameters of NYHA, LVEF, LVDd, MLHFQ QoL score, NT-proBNP levels and tolerated workload during stress testing in our cohort with recent-onset cardiomyopathy, the response rate was 100%. The gene discussed is NPPB; the disease is cardiomyopathy.