For the second year, the Rare Diseases Foundation was pleased to be able to launch, jointly with the AFM-Telethon(www.afm-telethon.fr), the call for projects "Innovative therapeutic proof of concept in rare diseases". This call for projects was born out of the desire to support innovative research projects related to the consolidation and maturation of therapeutic proof of concepts in rare diseases.
Today, there are more than 7000 rare diseases in France, affecting approximately 3 million people, 95% of whom remain untreated to this day. Given the great diversity of tissues and organs affected and the pathological mechanisms involved, the multiplication of innovative therapeutic approaches is a necessity. The clinical development and availability of new treatments for patients depends in particular on the quality of proof of therapeutic concepts.
The objective of this call for projects is to finance innovative projects for the consolidation and maturation of proof of therapeutic concepts supported by Rare Diseases Health Networks in order to develop new treatments for one or more groups of rare diseases.
The Rare Diseases Health Networks(www.filieresmaladiesrares.fr) are structures approved by the Ministry of Health and Research that bring together and coordinate a group of actors involved in rare diseases and their management. This call for projects is in line with axis 2 of the missions and objectives of the Rare Diseases Health Networks, set by the3rd National Plan for Rare Diseases, aiming to coordinate fundamental, translational and clinical research. It offers an opportunity for the Sectors to identify and monitor projects of interest for the themes they support and to identify new research teams working on their pathologies of interest.