ea0090p331 | Diabetes, Obesity, Metabolism and Nutrition | ECE2023
Haqq Andrea M.
, Chung Wendy
, Dollfus Helene
, Iqbal Anoop
, A. Martos-Moreno Gabriel
, Poitou Christine
, Yanovski Jack A.
, Malhotra Sonali
, Miller Paul
, Yuan Guojun
, Forsythe Elizabeth
, Clement Karine
, Argente Jesus
Background: In patients with Bardet-Biedl syndrome (BBS), signaling impairments in the melanocortin-4 receptor (MC4R) pathway lead to hyperphagia and severe obesity, which negatively impact quality of life (QOL). We evaluated the impact of setmelanotide, an MC4R agonist, on age-appropriate weight-related parameters, hunger, and QOL in a Phase 3 trial of patients with BBS to further characterize clinical benefit in this patient population.Methods: Patient...