However can see the progress made in twenty years

The 20th Telethon, held on 8 and 9 December, has raised 101,47 million in pledges despite the controversy, engaged in the days that preceded it, by some members of the Catholic Church about the use of a portion of the money raised to fund research on embryonic stem cells.

This record figure reflects the support each year renewed the public to the cause of the myopathes. Support to be put to the assets of the French Association against myopathies (AFM) because it is she who successfully through the Telethon, generate a spirit of solidarity around these patients previously excluded from society. Service of the AFM States confined to this, it's already considerable. But the association also played a very original role in two areas: biomedical research and the governance of the associations of disease control.

Concerning research in biology and medicine, the successive Généthon accompanied and stimulated its development. "The AFM has realized that only basic research would find solutions for the sick," observes Ketty Schwartz, President of the Scientific Council of the AFM. In fact, it is the alliance between the AFM and the Centre for the study of human polymorphisms, Genethon I, which led to the establishment of the first maps physical and genetic of the genome human, cards that have been used by Généthon 2 to identify and locate more than 100 genes of neuromuscular or genetic diseases. With Généthon, the AFM has made the choice risk gene therapy and established Cape Town, 1997, on the production of synthetic and viral vectors to transport up to the Interior of the nucleus of the cells, "gene drugs." Cell therapies then also benefited from its support, especially with the arrival of stem cells, including embryonic where critics.

Networks of expertise

At each step, with the financial windfall of the Telethon, the AFM was able to "dream big" and accompany, or even trigger for what is of the France, the industrialization of biology by the marriage of molecular biology, computer science and automation. In the same spirit, it has also funded the creation of various tools, whether it is Bank of DNA or animal models of diseases. Finally, it sparked the creation of networks of expertise and know-how to bring out the MYOLOGY (science of muscle) as a new discipline. "In many areas, observes Michel Callon, researcher at the Centre of Sociology of the innovation of the school of mines, the AFM thus prepared the way, turning the relay to public structures. As the national Centre of sequencing, the national Centre of genotyping or public research institutes. In this, the AFM played a role of general interest, beyond the sole cause of his patients.

Developing alliances with associations of patients suffering from rare genetic diseases, it also allowed for other "forgotten" to enjoy the fruits of the Telethon, as well as methods and approaches scientific and therapeutic similar. These approaches have also enabled progress on widespread diseases. Hence the concept of "disease model", that the AFM has highlighted to justify the use of large sums in research and treatment of rare diseases.

But it must also to the AFM the emergence of a new type of associations of patients, based on a partnership with the biological and clinical research. "The association has been able to find a balance between the autonomy indispensable to researchers and mentoring necessary to the achievement of its objectives", said Michel Callon. If it has a Scientific Council, it is indeed only a role of Council and proposal. It is the Board of directors only patients and their families who, as a last resort, made the choice and responsibility when it comes every year accountability to donors at the Telethon. "Finally, the AFM has participated in the emergence of a new way to research and innovation that involves all stakeholders, including beneficiaries, explains Michel Callon." Naturally, the association is not devoid of criticism, whether it be scientific choices and to exercise its power, both internal and external. However can see the progress made in twenty years. With a better support overall, the life expectancy of patients grew nine years on average. New challenges are waiting him yet, both scientifically and in ethics.