Designing Preclinical Studies to Robustly Inform Clinical Success in Alport Syndrome
- Leveraging highly translational models that closely mirror human Alport syndrome to enhance predictive value
- Testing therapies on top of standard of care by incorporating ACE inhibitors or ARBs to reflect real-world clinical settings
- Predefining clinically relevant endpoints by prioritizing GFR, albuminuria, and biomarkers or structural changes over retrospective data selection
- Focusing on GFR as the key outcome to enable robust longitudinal assessment and better predict clinical and regulatory success