3 results
Approved WMOCompleted
Primary:To assess the safety and tolerability of Firdapse after single and multiple doses Secondary:Part 1 only: To determine the dose-related PK profile of single, escalating doses of Firdapse;Part 2 only: To assess the steady-state plasma PK and…
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)