Patient and caregiver experiences with a patient-support program for setmelanotide treatment of patients with Bardet–Biedl syndrome

Abstract Background Bardet–Biedl syndrome (BBS) is a rare genetic disease caused by impaired cilium function and characterized by a plethora of symptoms, including hyperphagia and early-onset obesity, that negatively affect patient and caregiver quality of life. Here, we assessed real-world patient...

وصف كامل