Login / Signup

Neurofilament light chain levels correlate with clinical measures in CLN3 disease.

An Ngoc Dang DoNinet SinaiiRuturaj R MasvekarEva H BakerAudrey E ThurmAriane G SoldatosSimona E BianconiBibiana BielekovaForbes D Porter
Published in: Genetics in medicine : official journal of the American College of Medical Genetics (2020)
CSF and serum NEFL levels increase in multiple neurologic conditions. Here, we show that CSF and serum NEFL levels also increase in CLN3 (versus non-CLN3) and correlate with other disease-relevant measures. These findings suggest NEFL as a relevant and feasible biomarker for applications in CLN3 clinical trials and management.
Keyphrases
  • clinical trial
  • cerebrospinal fluid
  • randomized controlled trial
  • study protocol
  • phase iii