Why this disease?
Why this apparent resignation of the medical establishment?
After examining a child with reduced movements, how can a specialist condemn a child, admit that he cannot do anything, be incapable of making the correct diagnosis, of prescribing the correct equipment and adapted care in time?
No explanation, no drug, no hope.
In order to counter this unacceptable situation, a group of determined families decided to get involved in a common project in 1958 to change their fate, fight diseases, support research and find a cure as quickly as possible.
The solidarity of families progressively gave rise to a strategy: help patients affected by NMDs, understand the origin of the diseases and then try to find a cure.
As a result of an innovative strategy, of the results obtained in genetics and of the partnerships developed, AFM has become a major player in the fields of health economy and scientific and medical research .
It all started in 1986. Circumstances were such that Duchenne muscular dystrophy was the first genetic disease to be described. Following this discovery, families immediately asked Anthony Monaco ( the discoverer of the gene) two questions : how can you transform a faulty gene into a therapeutic gene ? How can you speed up the discovery of genes responsible for other neuromuscular diseases and also other genetic diseases?Since then, AFM has implemented a strategy designed to respond to these two questions. Suitable responses, however, require the implementation of a general strategy based on two types of diseases - genetic diseases and rare diseases - which include neuromuscular diseases. The objective is to reach a critical mass of knowledge, which is the only way to generate progress for patients. To reach this objective, AFM created Généthon, a hign-tech laboratory, in 1990.
The first strategic choice of AFM was thus to get involved in the field of genetics. The second was to conduct a global policy of genome exploration. This strategy bore fruit, since researchers all over the world now successfully use the tools created by AFM-Généthon.
The new therapies developed by the AFM thanks to Téléthon donations are today being tested on so-called “model” diseases, most of which are rare. As they are the first natural candidates for treatment, a quick and effective proof of concept of a therapy is made. Once over this stage, the therapeutic pathway tested can be applied to other (rare or frequent) diseases linked to the same physiological logic.
Thus, certain therapeutic pathways that have been developed can be applied to non-genetic diseases concerning the whole of the population.
Whether curing diseases or reducing disability, AFM keeps the same strategy : finding solutions, implementing them (alone or with partners), and after verifying their effectiveness, encouraging the government or institutional partners to participate so benefits can be shared.
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