IMPE Abstracts

Bardet-Biedl syndrome (BBS) is a rare disease associated with signaling defects in the melanocortin-4 receptor (MC4R) pathway that lead to hyperphagia, severe obesity, and, consequently, reduced quality of life (QOL). We investigated the impact of setmelanotide, an MC4R agonist, on weight, body composition, hunger, and QOL in a Phase 3 trial of patients with BBS to characterize clinically meaningful and patient-relevant benefit. Patients aged ≥6 years with BBS and obesity w...