3 results
Approved WMOCompleted
The objective of this study is to determine whether influencing the protein pathway thought to underlie TSC by Everolimus treatment will improve cognitive abilities in these children.
Approved WMOCompleted
The primary efficacy variable is the change in PV from baseline as assessed by 3 D ultrasonography after 78 weeks of double-blind treatment with OM 20-40 mg daily compared to ATE 50-100 mg daily.Secondary efficacy variables:Change from baseline PV…
Approved WMORecruiting
The primary objective of RIN-PF-303 is to evaluate superiority of inhaled treprostinil against placebo for the annual rate of change in absolute forced vital capacity (FVC) from baseline to Week 52.