The Leigh Syndrome International Consortium funds research to improve diagnosis, treatment, and care for Leigh syndrome. Across two grant cycles, the Consortium has:

  • funded 11 research projects globally.
  • invested $329,000 USD into Leigh syndrome research.
Previous recipients

2021: Massimo Zeviani, University of Padova, Italy
Gene therapy in mouse models of Leigh syndrome

2021: Michael Decressac, Université Grenoble-Alpes, France
Combining gene replacement and focused ultrasound to treat Leigh syndrome

2021: Dao-Fu Dai/Alessandro Prigione, University of Iowa, USA, Heinrich Heine University, Germany
Induced pluripotent stem cells (iPSC)-driven drug repositioning for Leigh syndrome

2021: Roan Louw, North-West University, South Africa
Using a variety of pre-clinical disease models to identify novel drug candidates for the treatment of Leigh syndrome

2021: Gino Cortopassi, University of California, Davis, USA
Safety, Efficacy and Mechanism of a Nicotinamidated Fumarate for Leigh syndrome

2019: Michael Palladino, University of Pittsburgh, USA
Novel dietary and pharmacological therapies in a Drosophila model of maternally inherited Leigh syndrome

2019: Gino Cortopassi, Regents of the University of California, USA
Fumarates for Leigh syndrome

2019: Alessandro Prigione, Heinrich Heine University, Germany
iPSC-driven repositioning of PDE5 inhibitors for Leigh syndrome patients carrying MT-ATP6 mutations

2019: Robert McFarland, Wellcome Centre for Mitochondrial Research, UK
Leigh syndrome: Investigating outcome measure & natural history (LION) - a prospective, longitudinal cohort study

2019: Dao-Fu Dai, University of Iowa, USA
The role of microglia, NAD+ and mitochondrial ROS in Leigh syndrome

2019: Brunella Franco, Fondazione Telethon, Italy
Therapeutic efficacy of miR-181a/b down regulation in Leigh syndrome