Psychometric evaluation of novel hyperphagia questionnaires in a real-world setting for patients with a rare MC4R pathway disease
The SoH: Caregiver and IoH: Caregiver demonstrated preliminary validity, reliability, and consistency in a real-world setting. Research is underway to further validate these measures for use in clinical trials for BBS and other MC4R pathway-related diseases associated with obesity.
Authors
- Caroline Huber (ORCID: https://orcid.org/0000-0003-3767-2719)
- Jeremy Pomeroy (ORCID: https://orcid.org/0000-0002-5270-9981)
- Ella Hagopian (ORCID: https://orcid.org/0000-0003-0506-7004)
- Andrea Maria Haqq (ORCID: https://orcid.org/0000-0002-6256-4982)
- Alexandra Greatsinger (ORCID: https://orcid.org/0000-0001-8576-8614)
- Usha G. Mallya
- Min Yang (ORCID: https://orcid.org/0000-0002-4864-6658)
Institutions
- Marshfield Clinic (US)
- University of Alberta (CA)
- Jazz Pharmaceuticals (United States) (US)
- Rhythm (United states) (US)
- Analysis Group (United States) (US)
- The University of Texas at Austin (US)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-08-22
- DOI
- https://doi.org/10.1186/s13023-025-03948-1
- Primary Topic
- Immunodeficiency and Autoimmune Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00