Krista Casazza talks about validating key biomarkers in Niemann-Pick type C and why they are essential for future clinical trials and regulatory approval. The discussion focuses on emerging candidates such as 24-hydroxycholesterol, neurofilament light chain, and calbindin-D, alongside the urgent need for data harmonisation and collaboration across the NPC community.

Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment

Krista Casazza, et al

https://doi.org/10.1002/jimd.70075