Review
Open access
Aug 2026
GBA1 mutations as a role model for precision medicine in Parkinson’s disease
This review critically examines the molecular and clinical spectrum of GBA1 -associated PD, highlighting the relationship between variant severity, lysosomal dysfunction, and disease progression, and summarizes emerging disease-modifying therapeutic strategies, including small-molecule GCase activators, pharmacological...
Christiane Oleksy, I. Boussaad, Z. Landoulsi et al.
· npj Parkinson's Disease · 0 citations