4 results
Primary ObjectiveTo evaluate the efficacy of lumacaftor in combination with ivacaftor throughat Week 24 in subjects with cystic fibrosis (CF) who are homozygous for the F508del CFTR mutation on the CF transmembrane conductance regulator (CFTR)…
primary objective: To evaluate the long-term safety and tolerability of lumacaftor in combination with ivacaftor in subjects with cystic fibrosis (CF), homozygous or heterozygous for the F508del-cystic fibrosis transmembrane conductance regulator (…
The main objective of the present study is to compare the clinical outcome of bone marrow stimulation alone versus bone marrow stimulation and bone marrow aspirate concentrate (BMAC) for small symptomatic osteochondral defects of the talus.
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…