MNGIE (Mitochondrial Neuro-Gastro-Intestinal Encephalomyopathy) treatment candidates have emerged, creating hope for clinical trials for patients affected by this condition. However, a comprehensive, up-to-date natural history study of MNGIE will be crucial to inform the design of these clinical trials and select appropriate clinical and biochemical outcome measures.
We encourage reserachers and patients to learn about and collaborate with the worldwide, collaborative study led by clinical researchers from the University of Cambridge (Dr. Jelle van den Ameele) and University of Bologna (Prof. Caterina Garone). The study is funded by Pierrepont Therapeutics Inc, and has received ethical approval from the University of Cambridge Human Biology Research Ethics Committee.
This study has been registered on ClinicalTrials.gov, with study number NCT07627217
If you have any questions, email add-tr.mitoteam@nhs.net