Research programme :

The AFM’s policy for the development of therapies is centred around three axes. The first is  fundamental research and its development – to help the progress of muscle knowledge, transfer or gene repair techniques as well as the use of differentiated cells or stems for therapeutic ends. The other two axes are: the application of techniques gained from knowledge about and diagnosis of genetic diseases and research applied in therapies (vectorology, cell therapy, development of animal models, and tools for evaluation and clinical trials in humans). Since 1989 the AFM has been engaged in using genes to attain its aim – cure. Ten years later, in 1998, it launched the “Great Attempt,” an ambitious five-year scientific programme aimed at demonstrating the feasibility of gene therapies. This challenge proved to be a success beyong all expectations, with the first victories in the form of therapies. In 2002 the AFM launched a new (five-year) challenge – “The New Frontier,” its aim being to increase the number of therapeutic trials in humans.
In 2007, the AFM supported nearly 400 projects coming from 4 calls for tender following the expert opinion by the Scientific Council’s committee of experts.
In 2006 the AFM carried out an in-depth reorganisation of its Scientific Council in order to reinforce its orientation towards clinical development.

In this context, Généthon changed its activities by turning to the development of gene therapy techniques. The Association had come to the conclusion that there were no suitable tools to implement the therapeutic strategies based on an understanding of the genes. These strategies are theoretically applicable in a large number of medical disciplines: genetic diseases, but also cancers, cardiovascular diseases, neurodegenerative diseases, auto-immune diseases, infectious diseases or even trauma cases.
For that reason, Généthon developed an original strategy combining basic research and pre-industrial development, with the design, development and production of vectors that could transport the gene-drug to the nucleus of cells.The Généthon III project was born. Généthon IV is the first laboratory in France authorised to produce AAV clinical vectors, and Généthon V is becoming a biotherapy company for rare genetic diseases.

The Great Attempt led to the development of tools, networks and research centres for gene-based therapies. The first successes in gene therapy confirmed that we made the right choices. Since further steps need to be taken towards a cure, AFM takes up a new challenge in 2002 : another five-year project called the '' New Frontier ''("la Nouvelle Frontière"), with an aim to increase the number of clinical trials in humans and implement gene-based therapies.

At the time when innovative therapies are first being tried out in humans, the AFM must face up to a new challenge – effectiveness. Effectiveness in the choice of programmes financed as well as in the therapeutic result for the patients included in the programmes. To attain this objective, during 2007 the AFM considerably increased its motor role in the initiation and support of clinical trial projects, while all the time ensuring a balance with fundamental research, necessary to the understanding of the mechanisms linked to neuromuscular diseases and to the development of therapeutic leads. This has been carried out in the context of international collaboration, essential in the field of rare diseases.