3 results
Approved WMOCompleted
Parts D and E: Primary Objectives:• To evaluate the safety and tolerability of VX-445 in TC with TEZ and IVA in subjects with cystic fibrosis (CF)• To evaluate the efficacy of VX-445 in TC with TEZ and IVA in subjects with CFSecondary Objectives• To…
Approved WMOCompleted
To assess the safety and tolerability profile of LJN452 and to determine the early hepatic response to different doses of LJN452 in patients with phenotypic non-alcoholic steatohepatitis (NASH). Data from this study will be used to support further…
Approved WMORecruiting
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.