Mitochondrial Neurogastrointestinal Encephalopathy (MNGIE) is an extremely rare genetic disorder and mitochondrial disease that affects the digestive and nervous systems. Given its rarity, the challenges in understanding and treating this condition are substantial.

 

Project COLT-MNGIE is promoted by the Mitocon patient advocacy group for people affected by mitochondrial diseases and Cochrane MS and Rare Diseases of the CNS. This project aims to develop a Core Outcome Set (COS) for MNGIE, in order to provide a consistent framework to evaluate the efficacy and safety of liver transplantation (LT) and other promising enzyme-replacement treatments for MNGIE.

 

The success of this project will significantly benefit MNGIE patients and their families by improving treatment standards and patient-centred outcomes.
 

We ask for your support in spreading the word about our online and completely anonymous survey.

 

This is the survey link, available in 5 languages: https://eu.jotform.com/form/240093045599360

 

Given MNGIE's rarity, we need to reach as many people as possible. Only in this way we can ensure to involve MNGIE patients and include the comprehensive representation of their needs and preferences.

 

To make it easier to share the survey with MNGIE patients, we have developed some explanatory slides, attached for your convenience. These slides provide a clear overview of the project and highlight why patient participation is essential: Info Survey for MNGIE Patients

 

Your help in disseminating this survey will be invaluable in advancing research and care for those affected by MNGIE!

 

We are fully available to address any question and concern at: cochrane.mito@ausl.bologna.it or scientific.office@mitocon.it.