70% of research into rare diseases in France is funded by AFM-Téléthon
Overall, half the trials supported by AFM-Téléthon involve non-neuromuscular rare diseases. Each of these trials is strategic because it will boost progress in developing treatments for other diseases.
Some of these rare diseases such as immunodeficiencies, diseases of the retina, skin or central nervous system, need local intervention (bone marrow, retina, etc.), leading to faster progress in solving scientific (immunology, vectorology, etc.), regulatory or production problems, and so on, and obtaining the first solid results demonstrating the efficacy of innovative treatments.
At the same time, AFM-Téléthon created the Biotherapies Institute for Rare Diseases, that gathers its four labs, in order to enhance the progress made by working together.
Furthermore, it is the main financier of the Rare Diseases Foundation. Creation of this Institute, brings together patient associations, ministries and public organizations, focusing on rare diseases, for the first time, in a joint project intended to encourage, develop, coordinate and streamline research into rare diseases. The GIS-Institut des Maladies Rares carries out strategic actions: a very high speed sequencing platform, ERDITI partnership with industry for screening molecules with a therapeutic goal.
The Association is also a member of the Imagine Foundation for scientific cooperation, which aims to improve diagnosis and treatment of genetic diseases by bringing together researchers, physicians and families at the Necker hospital (Paris).