Progress in the knowledge of molecular biology and of physiopathology now enables pharmacology to tackle the causes of diseases and no longer just the symptoms.
Thus, AFM supports a number of new avenues for the development of pharmacological treatments which may help slow down the course of neuromuscular diseases.
For the list of clinical trials, please consult the Clinical trials section.

AFM is also involved at the European level and supports the development of medicinal products for rare diseases.
For information about AFM’s action in this field, please click here. 

Many  pharmacological trials are under way this year (2008).
Certain of them concern molecules already used in other pathologies and which are being applied to rare genetic diseases: vitamin C for Charcot-Marie-Tooth disease, Bezafibrate for a metabolic myopathy, Riluzole for spinal amyotrophy etc. Others concern new molecules. For example TRO16922, developed by the company Trophos with the AFM’s support, is intended for the treatment of spinal amyotrophy and amyotrophic lateral sclerosis. The clinical development of this molecule took place through various studies. Results are pending in the study into the effect of Salbutamol in facio-scapulo-humeral muscular dystrophy, now at the end of phase III.