Arimoclomol Shows Potential to Aid Gaucher Patients in Phase 2 Trial
Arimoclomol, Orphazyme’s investigational therapy for Gaucher disease and other disorders, leads to clinically meaningful, dose-dependent reductions in liver and spleen size in patients with type 1 or 3 Gaucher, according to top-line results of a Phase 2 trial. Despite failing at its primary…