Publications by authors named "Gerrit Ruiterkamp"
Article Synopsis
- Mitochondrial disease is a rare condition currently lacking approved treatments, with sonlicromanol being a promising candidate that modifies key metabolic and inflammatory pathways.
- A Phase 2b study was conducted to evaluate sonlicromanol's safety and efficacy in adults with a specific genetic mutation, involving both a randomized controlled trial (RCT) and a long-term extension study.
- While the primary endpoint of the RCT didn't show significant results, there were indications of improvement in certain cognitive and emotional assessments among patients who were more affected at baseline.
Background: METHODS: The KHENERGYC trial will be a phase II, randomised, double-blinded, placebo-controlled (DBPC), parallel-group study in the paediatric population (birth up to and including 17 years). The study will be recruiting 24 patients suffering from motor symptoms due to genetically confirmed PMD. The trial will be divided into two phases.
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