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Lookup NU author(s): Dr Holly Mabillard, Professor John SayerORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© The Author(s) 2025. Alport syndrome (AS) is a progressive monogenic glomerular kidney disease characterised by kidney function decline, hearing loss, and ocular abnormalities, often leading to early-onset kidney failure (KF). While current therapies, such as renin-angiotensin system inhibitors (RASi), offer some benefits, many patients still experience KF at a young age, highlighting the need for additional treatment options. Glucagon-like peptide-1 receptor agonists (GLP-1 RAs) have emerged as promising agents with demonstrated cardiovascular and nephroprotective effects in type 2 diabetes (T2D) and chronic kidney disease (CKD) patients. Evidence from several major clinical trials has shown that GLP-1 RAs can reduce cardiovascular events and slow CKD progression by reducing albuminuria. Their potential mechanisms of action include anti-inflammatory, anti-fibrotic, and antioxidative effects, making them particularly relevant for the treatment of AS, where inflammation and fibrosis play crucial roles in disease progression. This review explores the therapeutic potential of GLP-1 RAs in AS, summarising pre-clinical and clinical data and elucidating the pathways through which GLP-1 RAs might offer renoprotective benefits. We advocate for further research into their application in AS and recommend the inclusion of AS patients in future clinical trials to better understand their impact on disease progression and patient outcomes.
Author(s): Boeckhaus J, Mabillard H, Sayer JA
Publication type: Review
Publication status: Published
Journal: Journal of Rare Diseases
Year: 2025
Volume: 4
Online publication date: 01/03/2025
Acceptance date: 17/12/2024
ISSN (electronic): 2731-085X
Publisher: Springer Science and Business Media Deutschland GmbH
URL: https://doi.org/10.1007/s44162-024-00065-8
DOI: 10.1007/s44162-024-00065-8
Data Access Statement: No datasets were generated or analysed during the current study.
