2 results
Approved WMOCompleted
Study Rationale and ObjectivesThe objective of the study is to determine the efficacy and safety of lacosamide, a sodium channel blocker, in patients with pain due to SCN9A-associated SFN. The proposed study plans to recruit patients with clinically…
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).