2 results
Approved WMOCompleted
The study will compare the overall survival (OS) between the MABp1 treated and placebo arms. Secondary endpoints will include change in lean body mass from screening to the cycle 5 assessment and change in quality of life.
Approved WMORecruiting
Primary Objective:To evaluate the effect of sonlicromanol on motor symptom severity in children with genetically confirmed mitochondrial disease affecting oxidative phosphorylation during a 6 month treatment period (GMFM).