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Clinical trials supported by AFM
At the present time the AFM is contributing financially to 34 clinical trials – either ongoing or in preparation. They concern both neuromuscular and non-neuromuscular diseases :
- neuromuscular diseases
- neurological or neurodegenerative diseases: leukodystrophies (2 different forms), Huntington disease, Friedreich ataxia and San Filippo diseases (2 different types)
- blood diseases: drepanocytosis, beta-thalassemia and porphyria
- immune deficiencies: ADA and WASP
- skin disease: epidermolysis bullosa (2 different forms)
- eye disease: Leber Amaurosis
- 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.
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