While clinical trials with hepcidin mimetics have been prematurely terminated due to the absence of efficacy (molecule La Jolla Pharmaceutical Company-401, NCT03381833; molecule PTG-300, TRANSCEND trial, NCT03802201), it remains to be determined whether molecules regulating hepcidin expression or ferroportin function are more effective at improving anemia in thalassemia patients. The gene discussed is HAMP; the disease is anemia (phenotype).