Robust outcome measuresThe ultimate goal of this research group is to select the most robust outcome measures to follow the health status of patients. Robust outcome measures are essential to monitor the natural disease course and to explore the effect of interventions.
AimsOur three research aims to select precise and clinically relevant outcome measures to monitor the wellbeing of patients, both children and adults, with mitochondrial disorders.
- We aim to test the value of existing instrument
- We aim to develop and validate new tests
- We aim to select the most appropriate instruments for natural history studies and clinical trials
We have validated the Gaitrite as a suitable instrument for the quantification of gait. Many instruments are currently under evaluation.
Existing instrumentsWe have validated the Gaitrite as a suitable instrument for the quantification of gait in children and adults with mitochondrial disease. Many instruments, including the 6-minute walking test, questionnaires for wellbeing, accelerometry, the PRPP, and timed tests are currently under evaluation.
We have developed the International Paediatric Mitochondrial Disease Scale. Also, a 6-minutes mastication test was developed.
We’ve recently completed a validation study in children with mitochondrial myopathy.