Patient and caregiver experiences with a patient-support program for setmelanotide treatment of patients with Bardet–Biedl syndrome
Based on this real-world survey of patients with BBS and their caregivers, setmelanotide improved key symptoms related to insatiable hunger and obesity. Personalized multidimensional nursing support at the start of treatment can help address unmet support needs in BBS and may contribute to high rates of treatment satisfaction and adherence.
Authors
- Lars Pape (ORCID: https://orcid.org/0000-0002-3635-6418)
- Metin Cetiner (ORCID: https://orcid.org/0000-0002-0918-9204)
- Ilja Finkelberg
- Anja Gäckler (ORCID: https://orcid.org/0000-0002-3630-4386)
- Tom Hühne
- Pia Brensing (ORCID: https://orcid.org/0009-0005-7965-8888)
- Johannes Jaegers
- Ioanna Maria Polichronidou
- Sinem Karaterzi
Institutions
- Essen University Hospital (DE)
- University of Duisburg-Essen (DE)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-06-08
- DOI
- https://doi.org/10.1186/s13023-025-03835-9
- Citations
- 6
- Primary Topic
- Genetic Syndromes and Imprinting
- Type
- article
- Field-Weighted Citation Impact
- 10.90
Funders
- Universitätsklinikum Essen