Introducing SynLeigh: a new project on treatment development for Leigh Syndrome

4 Nov, 2026

Coordinated by Professor Alessandro Prigione and funded through the European Rare Diseases Research Alliance (ERDERA), SynLeigh will study sildenafil and cannabidiol (CBD) in laboratory models. The research aims to gather evidence to support preparations for future clinical studies into Leigh Syndrome.

An international working group will bring patient and family experiences into the project from its earliest stages. Members will help identify the symptoms, everyday challenges and improvements that matter most, while advising on treatment considerations and information.

We are inviting people to join the working group. No scientific knowledge is needed, and people can contribute according to their availability. Joining the group does not involve receiving or testing a treatment.

To introduce the project, an online webinar, “Introducing SynLeigh”, will take place on 7 December 2026 at 5pm CET. Researchers will explain their work, answer questions and discuss how patients and families can get involved.

The webinar will be held in English, with translation potentially available. Patients, families and researchers are encouraged to attend.

Visit the SynLeigh webpage to register for the webinar, explore the programme or contact IMP about joining the working group.