A phase 3 nonrandomized trial followed in which slightly larger numbers of patients, including both children aged ≥6 years and adults, with POMC, PCSK1, and LEPR deficiency were treated, resulting in a mean decrease of -25.6% ± 9.9 in patients with POMC or PCSK1 deficiency and -12.5% ± 8.9 in patients with LEPR deficiency (39). The gene discussed is PCSK1; the disease is hyperinsulinemic hypoglycemia, familial, 4.