Addressing an important challenge faced: Why over-reliance on rare genetic mutation models is misleading sporadic CNS drug discovery
05 May 2026
Disease Models
- How does reliance on rare genetic mutation models limit our understanding of the aging-driven, multifactorial mechanisms that underlie sporadic neurodegenerative diseases?
- To what extent do accelerated pathology and protein overexpression in genetic models produce misleading efficacy signals and biomarkers that fail to reflect true disease progression in sporadic patients?
- Does success in familial mutation models create false confidence in target validity, and how might this contribute to the high failure rate of CNS drugs in late-stage clinical trials?
Industry Expert
