Patient and caregiver experiences with a patient-support program for setmelanotide treatment of patients with Bardet-Biedl syndrome
Orphanet J Rare Dis. 2025 Jun 8;20(1):290. doi: 10.1186/s13023-025-03835-9. ABSTRACT BACKGROUND: Bardet-Biedl syndrome (BBS) is a rare genetic disease caused by impaired cilium function and …