Arimoclomol as a potential therapy for neuronopathic Gaucher Disease
Cathrine K. Fog, Paola Zago, Erika Malini, Lukasz M. Solanko, Paolo Peruzzo, Claus Bornæs, Raffaella Magnoni, Nikolaj H.T. Petersen, Bruno Bembi, Andrea Dardis, Thomas Kirkegaard
This study shows arimoclomol improves GCase function in GD models by enhancing its folding, maturation, activity, and localization.
bioRxiv · Mar 15, 2018 · PreprintNeuroscience