Longitudinal Biomarker Analysis in Neuronal EVs and Plasma Following GHF-201 Treatment in Adult Polyglucosan Body Disease Patients

Bidoki, S., Siddique, I.A., Mishra, K., Khoury, S., Mordechai, A., Lossos, A., Zaitlen, N., Kakhlon, O., and Bitan, G.