"Model" rare diseases
Neuromuscular diseases are very complex because they can affect the whole muscle mass as well as nerve cells. To confirm the pertinence of innovative pathways such as gene therapy or cell therapy as quickly as possible, AFM-Téléthon funds projects concerning less complex rare diseases which affect only a single organ or tissue such as the eyes, kidneys, immune system or skin.
Partnerships with other associations
Of the human trials supported by AFM-Téléthon, half of them concern rare diseases other than neuromuscular diseases. AFM-Téléthon funds some of them in partnership with other associations, such as Vaincre la Mucoviscidose (French Cystic Fibrosis Association), Rétina France or the Fondation du Rein (French Kidney Foundation), the Association pour l'Information et la Recherche sur les Maladies Rénales Génétiques (Association for Information and Research into Genetic Kidney Diseases), the Fondation d'aide à la recherche sur la sclérose en plaques, Arsep (Foundation for Research Assistance on Multiple Sclerosis).
Prospects for common diseases
Research on new treatments which the association supports is also paving the way for more common diseases. For example, one of the trials it supports concerns the treatment of myocardial infarction using stem cells. Similarly, the drug for progeria, a very rare disease which causes accelerated ageing in children, could help combat one of the side effects of triple therapies used to treat AIDS: premature ageing of the patient's cells. Finally the work done by I-Stem (laboratory co-founded by AFM-Téléthon) in creating skin from stem cells could be used to treat patients with severe burns.