SynLeigh: together towards a treatment for Leigh syndrome

An exciting international research project bringing researchers, patients and families together with the hope of moving closer to an effective treatment for Leigh syndrome.

IMP is delighted to be part of SynLeigh, alongside our patient organisation partners, Mitocon and Cure Mito. Together, we want to ensure that the experiences and priorities of people living with Leigh syndrome and their families help guide the research from its earliest stages.

We are looking for people living with Leigh syndrome and family members to join an international working group and help shape this research. No scientific knowledge is needed. Your experience is what matters.

Invitation: Our introductory webinar is an opportunity to learn about the project, meet the researchers and find out how you can get involved (read more below). 

What is SynLeigh?

SynLeigh is an international research project coordinated by Professor Alessandro Prigione and funded through ERDERA (the European Rare Diseases Research Alliance). It brings together scientific partners, including the Carlo Besta Neurological Institute in Milan, and Patient Organisations, IMP, Mitocon and Cure Mito.

Researchers will study sildenafil and cannabidiol (CBD) in laboratory models of Leigh syndrome, including forms associated with the SURF1 and NDUFS4 genes. The aim is to gather evidence to help prepare future clinical studies.

We hope this work will bring us closer to an effective treatment, with patients’ and families’ priorities helping to guide its development.

Help shape the research: join the working group

What would a meaningful improvement look like for you or your family? What should researchers understand about everyday life with Leigh syndrome?

Through the international working group, you could help researchers to:

• Understand the symptoms and everyday difficulties that have the greatest impact on patients and families.
• Identify which improvements would make the most meaningful difference.
• Consider practical aspects of a future treatment, such as how it is given and the monitoring it requires.
• Recognise needs and barriers when planning future clinical studies.
• Make information about the project clear and useful for the community.

Your input will help describe what a future treatment should offer from the perspective of patients and families. Joining the working group does not involve receiving or testing a treatment.

Take part in a way that works for you

You do not need scientific knowledge to contribute. We want to hear about your experiences, questions and priorities.

We understand that family commitments can make attending meetings difficult. You can contribute according to your availability, including by sharing questions or reflections when you cannot attend.

International meetings will be in English. Please tell us about any language or accessibility needs, so we can discuss what would help you participate.

If you would like to join the working group, or simply find out more, we would love to hear from you.

For Patient Organisations

Please help us reach people living with Leigh syndrome and their families by sharing this page and the webinar invitation with your community.

Experiences from different countries will help researchers understand the needs and priorities of our international community. Contact IMP if your organisation would like to help connect interested patients and families with the project.

Join our webinar: Introducing SynLeigh

7 December 2026 | 5pm CET 
Online | In English (Translation may be available)

Discover what SynLeigh hopes to achieve and how patients and families can contribute.

The researchers leading the different parts of the project will introduce their work and answer questions. 

Join us to:

• Learn about the project and its research plans.
• Hear how patient and family experiences can help guide the work.
• Ask questions and find out about the working group.

Looking ahead

Further webinars are planned for 2027, exploring the research on sildenafil and CBD, patient priorities and preparations for possible future clinical studies. Educational activities and an interactive session for patients and families are also being planned alongside the Mito Conference in Rimini, Italy, on 1–3 October 2027.

We will share more details as plans are confirmed.