International Journal of Molecular Sciences
Development of Small-Molecule Allosteric Modulators of Beta-Galactosidase (β-Gal) for the Treatment of GM1 Gangliosidosis and Morquio B
Small Methods
Characterization of Ligand Binding in Human Serum Albumin from Atomistic Energy Transfer Simulations
Movement Disorders
Glucocerebrosidase Target Engagement and Therapeutic Plasma and Cerebrospinal Fluid Levels After GT‐02287 Administration in Healthy Volunteers
Nature Partner Journals
A community-led initiative to de-risk and advance Parkinson’s disease therapeutic targets
Scientific Reports
A novel allosteric GCase modulator prevents Tau accumulation in GBA1WT and GBA1L444P/L444P cellular models
International Journal of Molecular Sciences
Allosteric Modulation of GCase Enhances Lysosomal Activity and Reduces ER Stress in GCase-Related Disorders
Journal of Medicinal Chemistry
Use of the Novel Site-Directed Enzyme Enhancement Therapy (SEE-Tx) Drug Discovery Platform to Identify Pharmacological Chaperones for Glutaric Acidemia 1