20 years and a few figures. In 2005, 104 million euros were collected in France and 1.8 million in the Alpes-Maritimes. 250 families rely on AFM support in the department. Out of every €100 spent by the AFM, €18.50 is allocated to collection and administration costs, and €81.50 to missions, with two-thirds devoted to research and one-third to social support for patients and families. The AFM maintains transparency. The French Muscular Dystrophy Association, over two decades, has gained the trust of the French public and the State, which has recognized the association as being of public utility.
Laurence Tiennot sets the stakes: “We need to provide the means to identify the genes responsible for all genetic diseases, and currently two out of three diseases have not been identified. Généthon has saved time in genetics and the last twenty years of research have provided tools to place markers on the human genome.” The other works and priorities of the AFM are set at the level of research on rare diseases, aided by a National plan: “Between 6,000 to 8,000 rare diseases are recorded. What is sometimes forgotten is that they affect three million French people,” notes Laurence Tiennot. The Association places great hope in the Institute of Myology at the Pitié Salpêtrière. Studying muscles and their cells leads to the identification of many proteins, causes of diseases, and targets for treatments. Dr. Sabrina Sacconi, a neurologist at the Neuromuscular Disease Reference Center at CHU in Nice, is part of a team conducting a therapeutic trial for Facioscapulohumeral Muscular Dystrophy for another year and a half. She explains the operation in simpler terms: “We cultured unaffected muscle cells to re-inject them into affected areas. It is a rather difficult injection technique. The experiment will be conducted on nine patients (three groups of three). The first injection is scheduled for November 9.” In Nice, professors Guerrino Meneguzzi and Jean-Philippe Lacour are working on therapeutic prospects for a hereditary skin disease: junctional epidermolysis bullosa (skin that peels). These costly studies are proof that progress is being made, thanks to donations, the Telethon, and the AFM. “Therapeutic trials represent a challenge, but the closer we get to drugs for treatment and cure, the higher the costs. The price of a drug can reach 800 million euros.”
Finally, other actions concern patients with care to increase both their life expectancy and quality of life. The AFM acts, not only during the Telethon event, on the citizenship of disabled people. Saïda Aouragh, a 25-year-old from Vence, shares her testimony which surpasses any speech about the AFM’s beneficent action: “The Association was my first moral support, especially in accepting the disease. There are three of us in the family affected by the disease. They helped us adapt the house. It’s a relief. I had to fight for schooling. I didn’t want to go to a specialized institution, but the college in Vence wasn’t equipped. I suffered as I could. In the end, we managed to get the necessary adjustments (sanitary facilities and elevator) and even though I didn’t benefit from them, my little brother did. Thanks to the AFM, I didn’t have to pay for my wheelchair. I am currently working on getting a specialized vehicle.” Saïda and the research conducted in the department illustrate the utility of every 3637 dialed on a phone and donations throughout the year.
