Votre navigation :

Clinical trials supported by AFM

At the present time the AFM is contributing financially to nearly 40 clinical trials – either ongoing or in preparation. They concern both neuromuscular (approx 50%) and non-neuromuscular diseases :
- 11 neuromuscular diseases
- 4 neurological or neurodegenerative diseases: adrenoleukodystrophy, Huntington disease, Friedreich ataxia and San Filippo disease (2 different types)
- 3 blood diseases: drepanocytosis, beta-thalassemia and porphyria
- 2 immune deficiencies: ADA and WASP
- 1 skin disease: epidermolysis bullosa (2 different forms)
- 1 eye disease: Leber Amaurosis
- 1 ageing disease: progeria
- myocardial infarction

These trials are distributed between :
gene therapy (50%) to correct genetic mutations and produce missing or deficient proteins
cell therapy to regenerate diseased tissue (20%)
classic pharmacology (30%) with molecules already used for other diseases but likely to be beneficial for neuromuscular diseases, or new molecules likely to attack directly the cause or the consequences of genetic damage.






Update 2008/07/07
A.F.M. - 1, rue de l´Internationale - BP 59 - 91 002 Evry cedex - Tél. : +33.1.69.47.28.28
Website realized by Ecedi.