Neurofilament light is a treatment-responsive biomarker in CLN2 disease.
Yuanbin RuCarley CoradoRussell K SoonAndrew C MeltonAdam HarrisGuoying K YuNancy PryerJohn R SinclairMartin L KatzTemitayo AjayiDavid JacobyChristopher B RussellSanjay J ChandrianiPublished in: Annals of clinical and translational neurology (2019)
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.