AFM’s accomplishments in the scientific field result from an efficient strategy geared toward curing neuromuscular diseases.
It has created a critical mass of knowledge that benefits more common disorders, thereby bringing neuromuscular diseases and rare disorders out of oblivion.
It has contributed to the rapid development of skills and technological tools that put therapeutic results for neuromuscular diseases within reach and raise patients’ hopes It has developed scientific tools to increase knowledge, knowledge that is vital for the development of new therapies. DNA banks, the maps of the human genome, and the discovery of a large number of genes are major advances originating from this strategy.
Using the same approach, it launched la Grande Tentative ("the Great Attempt"), a far-reaching scientific program of national and international collaborations for the development of gene-based therapies, and has recruited public and private partners.
After five years dedicated to the development of scientific tools and to the creation of infrastructures, AFM launched the New Frontier program (« La Nouvelle Frontière »). The objective is to increase the number of clinical trials in humans.
The Association’s involvement in the fight against neuromuscular diseases has led it to explore all potential therapeutic pathways : therapeutics will be different for each pathology and it has become evident that, for the same pathology, solutions will be different depending on the patients and on the type of genetic mutation. As a result, the Association encourages all new initiatives coming from laboratories (pharmacological treatments, gene and cell therapies). It supports an increasing number of meetings between patients, researchers and clinicians driven by the same conviction that sharing contributes to finding new answers to these questions. Therefore, out of clinical trials planned by year 2007, some involve neuromuscular diseases and others rare « model » genetic diseases for which potential treatments are the most advanced (blood and skin diseases, neurological diseases). By investing in these clinical trials, AFM pursues the same objective : to develop innovative therapies which will provide a treatment for neuromuscular diseases. Past successes have demonstrated that this objective will benefit many other diseases as well.
From 1988 to the end of 2007, AFM invested 845 million euros for curing diseases (66 million euros in 2007).