The Lily Foundation
Professor Grainne Gorman, Professor Robert McFarland, Professor Robert Taylor, Dr Caterina Garone and Dr Youcef Mehellou
The University of Newcastle, University of Cambridge and Cardiff University
Towards devising a therapeutic strategy for patients with recessive RRM2B related mitochondrial disease.
While pharmaceutical treatments for different types of mitochondrial disease (mito) are emerging, there are few effective therapies. This project will facilitate research into treatment for recessive RRM2B related mitochondrial disease (a specific form of mitochondrial DNA depletion syndrome) with the aim of developing disease-specific therapeutic strategies and the design of future clinical trials.
The project will be conducted by a close collaboration of clinicians, scientists, pharmacists, allied health professionals (including a highly experienced physiotherapist) and a project manager across three centres (Newcastle, Cambridge and Cardiff), with diverse expertise in therapeutic development, mitochondrial medicine and patient-centric trial design.