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The AFM-Téléthon is an association created in 1958 by patients and their parents who were suffering from rare genetic diseases: neuromuscular diseases. AFM-Téléthon, which has been organising the French Telethon since 1987, has the firm conviction that only by concentrating resources, will it be able to obtain significant results. That is the reason why it has implemented a strategy that includes problems shared by all rare diseases to benefit all diseases.
AFM-Téléthon is a key player in research and development of innovative therapies for rare diseases and the struggle for citizenship of those who suffer from them:
- 4 research centres who are leaders in their fields: Genethon and Atlantic Gene Therapies for genetic therapy of rare diseases; the Myology Institute for muscles; I-stem for stem cell research and its application to rare diseases. These four laboratories gathered at the institute of biotherapies. With its 650 experts, it represents a unique strike force against rare diseases, from research to clinical development.
- Founding member of the Foundation for Rare Diseases. By and large, AFM-Téléthon supports 70% of research on rare diseases in France (source: Leem).
- Development of innovative therapies for rare diseases: about 30 different diseases currently ready for medication through 36 clinical trials supported by AFM-Téléthon thanks to Telethon donations (neuromuscular diseases, neurological diseases, diseases of the blood system, vision, skin, liver, immune system, etc.). The emergence of new types of therapies for rare diseases has allowed new tools and new approaches to better combat all of these types of diseases. Rare diseases are the medical innovation laboratory that benefits the greatest number of patients.
- We support the Rare Diseases Platform and its entities. We carry out actions to assist those in our government in recognising and intensifying the combat against rare diseases.
- Social and technological innovation in support of autonomy of those who are handicapped; actions to claim that rights set forth in the 2005 French law be respected.
Thanks to Telethon donations, AFM-Téléthon can boast of being at the origin of key advancements for curing rare diseases:
- The first human genome maps written by the Genethon Laboratory which was created by AFM-Téléthon and funded by Telethon donations. France has thus become the global leader in genetic research!
- The acceleration of the discovery of disease-causing genes.
- A more precise diagnostic and more targeted care for those suffering from these diseases has translated into much higher quality and a longer life expectation.
- The first therapeutic victories for children suffering from immunitary depression and rare brain or blood diseases.
- Genethon Bioprod, the leading production centre in the world for genetic therapy mediation in phase I /II for rare diseases.
- The creation of the Rare Diseases Hub, a resource centre that is unique in Europe and that brings together all key stakeholders for rare diseases.
- Rare diseases have emerged from oblivion and have become one France’s national health priorities: two national plans were obtained because of the support of associations.
- European recognition of rare diseases: in 1999, European rules allowed market authorisation for over 60 drugs targeting orphan diseases, in 2009, a European recommendation was adopted for rare diseases.
- Legislation for new rights for people suffering from handicaps (the right to receive compensation, the right to go to school, the right to receive training and be employed) in the 2005 law for handicapped people.