Endocrine Abstracts

Background: Patients with Bardet-Biedl syndrome (BBS), a rare syndrome associated with obesity and hyperphagia, commonly manifest traits of metabolic syndrome, including abdominal obesity, impaired fasting glucose, low high-density lipoprotein cholesterol, hypertriglyceridemia, and hypertension. Setmelanotide treatment results in significant improvement in weight, hunger, and quality of life in these patients and may improve measures associated with metabolic syndrome developm...

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...