Téléthon 2008 :

The French Téléthon is organised by the AFM (French Muscular Dystrophy Association).
This is a unique event – all together, wherever we are and whatever our background, once a year we take up this exceptional challenge for the medicine of the future!
Let’s join forces and win this combat against disease on the website http://donate.telethon.fr/ !

We are the force behind patients and families, behind researchers and physicians !

The combat against genetic diseases is entering a new era, that of trials in humans and the first therapies. These preliminary realisations have come after years of research thanks to the mobilisation of Téléthon organisers and supporters. At present, about thirty AFM-supported clinical trials for 29 different diseases are under way or in preparation. Confronted by diseases which cause children to lose their sight or the ability to walk, which isolate them from the outside world or make them age prematurely, families are fighting against fatality and researchers and physicians are committed to helping them succeed.

Children without immune defence are condemned to live in a bubble, cut off from the outside world. Thanks to cooperation between the French and Italian Téléthons, ten “bubble babies” have been cured and the treatment is about to be launched on the European market.

From childhood, this progressive disease attacks all the muscles. But hope is there – thanks to Téléthon support, a gene surgery trial on young patients is under way.

In one year, they age more than ten years. Fifteen European children affected with progeria should soon be able to participate in a treatment trial – developed by a team from Marseille – only five years after the discovery of the gene at the origin of the disease. This extremely rapid advance also opens the way to a better understanding of the normal mechanisms of ageing.

After restoring sight to blind dogs, a gene therapy laboratory team in Nantes, financed by the AFM, is tackling a rare genetic disease that causes affected children to lose their sight irreparably. This therapy also represents a hope for the treatment of age-linked macular degeneration, the main cause of blindness in the over-fifties.