2 results
Approved WMOCompleted
Primary: To assess the efficacy of GNX compared with PBO, as adjunctive therapy for the treatment of primary seizure types in children with genetically-confirmed PCDH19-related epilepsy during the 17-week double-blind (DB) phase. Secondary: • To…
Approved WMORecruiting
To assesses the efficacy of Tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1.