3 results
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).
This research project is divided into two projects. The first part of the study (Study 1) aims to find an answer to the question whether and to what extent biofeedback is a user-friendly and feasible intervention. In the main study (Study 2), we…
The study*s primary objective is to assess the safety and tolerability of CP-506 alone or in combination therapy. The 3 different modules explore the administration of CP-506: alone (Module 1) or in combination with Carboplatin (Module 2) or in…