The CNL3 Staging System for clinical research in Batten disease

Takeaway

  • The study developed a disease-specific staging system for CLN3 disease, a neuronal ceroid lipofuscinosis, which can effectively separate individuals into four distinct stages of disease.

Why this matters

  • The CNL3 staging system (CLN3SS) could potentially be used for the stratification of cohorts in future clinical trials.

  • Improvements in the design of CNL3 clinical trials have become increasingly important, following the recent development of a potential disease-modifying therapy.