The French Muscular Dystrophy Association (AFM-Telethon) is an association of activists, patients and parents of patients, affected by rare, progressive and significantly disabling genetic diseases, known as genetic neuromuscular diseases. It was created out of a conviction and a desire, to cure diseases that were long considered to be incurable. AFM-Telethon is also the Telethon, a fundraising event which brought rare diseases out of scientific and medical obscurity and triggered a three-fold revolution: genetic, social and medical.
Creation of the Association
In 1958, a handful of parents revolted against the ignorance and powerlessness of medicine and science in the face of the neuromuscular diseases affecting their children, decided to form the French Muscular Dystrophy Association (AFM-Telethon). Yolaine de Kepper, a mother of seven children, four of whom suffered from Duchenne muscular dystrophy (DMD), was the Association’s founder and first Chair.
AFM-Telethon fights to recognize diseases neglected by public authorities and poorly understood by physicians and researchers.
In 1976, the Association was awarded state-approved charity status.
The fight for civil status and autonomy
- In 1969, treatment for myopathy was 100% covered by French national health insurance.
- In 1972, AFM-Telethon introduced the first electric wheelchairs in France and campaigned for them to be covered by French national health insurance (which happened in 1977).
The fight to bring people together
In the 1980s, myopathy started to emerge from the shadows.
- In 1981, AFM-Telethon created its first scientific advisory committee. This was the beginning of a novel partnership between patients, researchers, and doctors.
- In 1986, during the Association’s second academic conference, a young researcher announced that they had discovered the gene responsible for Duchenne muscular dystrophy. This discovery was a pivotal one for AFM-Telethon, which then decided that genetic research would be the lynchpin of its commitment to finding a cure.
In order to have the financial resources to fund innovative and powerful research, in 1987 AFM-Telethon created the first French Telethon. It was an immediate success!
The fight for the cure
Thanks to the unprecedented support of French people during the Telethon, the families’ fight began to change shape.
AFM-Telethon created Genethon, a pioneering genetics and gene therapy laboratory for rare diseases. From 1992 to 1996, Genethon published the first maps of the human genome and made them available to the scientific research community. This was the starting point for the decoding of the human genome completed in 2003.
In the 1990s, AFM-Telethon set itself an incredible challenge, namely to use genes as medication to cure incurable diseases. In 2000, the French team of Prof. Alain Fischer (Necker Children’s Hospital), with support from the Telethon, celebrated the world’s first successful use of gene therapy for children suffering from SCID, or bubble babies, increasing AFM-Telethon’s determination to support the development of innovative therapies. Since 2007 we have seen a great many successes in rare diseases of the immune system, blood, brain, vision, and more recently, muscles.
Today a true revolution in medicine is underway worldwide. It is changing the world of rare diseases and saving the lives of children who would otherwise not survive.