Movement disorders : official journal of the Movement Disorder Society
Glucocerebrosidase Target Engagement and Therapeutic Plasma and Cerebrospinal Fluid Levels After GT-02287 Administration in Healthy Volunteers.
Raffaella Pozzi, Michele De Sciscio, Manuela Bosetti, Sara Cano-Crespo, Joanne Taylor, Terenzio Ignoni, Jonas Hannestad
Published: 202510.1002/mds.70111
Abstract
BACKGROUND: Variants in the GBA1 gene can increase the risk of Parkinson's disease (PD) by reducing glucocerebrosidase (GCase) activity, disrupting lysosomal and mitochondrial function, and increasing alpha-synuclein aggregation. The molecule GT-0228…
Preview only. Read the full abstract at the source