Neurofilament light chain in cerebrospinal fluid as a novel biomarker in evaluating both clinical severity and therapeutic response in Niemann-Pick disease type C1

Neena Agrawal, Nicole Y. Farhat, Ninet Sinaii, An Dang, Changrui Xiao, Elizabeth Berry‐Kravis, Simona Bianconi, Ruturaj Masvekar, Bibiana Bielekova, Beth Solomon, Forbes D. Porter