LAUSR

In the field of incurable neuromuscular diseases, the access to therapeutics is becoming a reality for some of them and some trials are on the way for the others. More precisely, all recent studies show the more effectiveness of these innovative therapies if administered early or pre-symptomatic stage of these diseases. For example, infant spinal muscular atrophy (SMA) is the most advanced with AMM treatment, several others, including gene therapy, in clinical trials, and three trials of pre-symptomatic administration. This makes this new-born screening a major public health issue with a diagnosis using molecular biology tools. In this context, the French Muscular Dystrophy Association (AFM-Telethons) and the French muscular disorders network (FILNEMUS) with others associations and partners want to be a force for proposals in this area. Speakers: Vincent Laugel CHU Strasbourg Christian Cottet Général Director AFM Téléthon Jean-François Malaterre Vice Président AFM-téléthon Brigitte Chabrol AP-HM Marseille