3 results
Approved WMOCompleted
Primary objectiveThe primary objective of the study is to determine the effect of lucerastat on neuropathic pain in subjects with Fabry disease (FD).Secondary objectives* To determine the effects of lucerastat on gastro-intestinal (GI) symptoms (…
Approved WMORecruiting
To evaluate the efficacy of PTC857 in reducing disease progression in subjects with amyotrophic lateral sclerosis (ALS)
Approved WMOPending
To investigate the reliability and feasibility of PK-guided prophylactic dosing of factor concentrates in hemophilia A and B patients (predictive performance).