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...

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Bibliographic Details

Published in:	Orphanet Journal of Rare Diseases
Main Authors:	Ilja Finkelberg, Ioanna M. Polichronidou, Tom Hühne, Pia Brensing, Sinem Karaterzi, Johannes Jaegers, Anja Gäckler, Lars Pape, Metin Cetiner
Format:	Article
Language:	English
Published:	BMC 2025-06-01
Subjects:	Bardet–Biedl syndrome Patient experience Caregiver experience Real-world evidence Melanocortin-4 receptor Setmelanotide
Online Access:	https://doi.org/10.1186/s13023-025-03835-9