Importantly, due to the involvement of PHIP in the leptin-melanocortin pathway, affected individuals with CHUJANS who suffer from therapy resistant obesity can participate in a clinical trial with an anti-obesity drug (setmelanotide (MC4R-agonist), ClinicalTrials.gov identifier: NCT04963231). The gene discussed is LEP; the disease is obesity due to melanocortin 4 receptor deficiency.