Neurofilament light is a treatment-responsive biomarker in CLN2 disease.

Yuanbin Ru,Carley Corado,Russell K Soon,Andrew C Melton, Adam Harris,Guoying K Yu,Nancy Pryer,John R Sinclair,Martin L Katz,Temitayo Ajayi,David Jacoby,Chris B Russell,Sanjay Chandriani

Annals of clinical and translational neurology（2019）

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摘要

Our data indicate that circulating neurofilament light is a treatment-responsive biomarker in CLN2 disease and could contribute to understanding of the pathophysiology of this devastating pediatric disorder.

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