Biomarkers in Niemann-Pick type C: Preparing for Clinical...

What this episode covers

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

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Frequently Asked Questions

How long is this episode of JIMD Podcasts?

This episode is 25 minutes long.

When was this JIMD Podcasts episode published?

This episode was published on December 2, 2025.

What is this episode about?

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...

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