> With the progression of knowledge in molecular biology and physiopathology, pharmacological methods can now be used to tackle not only the symptoms of the diseases, but their causes. For further information, click on pharmacological treatments.

> In the case of genetic diseases, gene therapy consists of replacing the function of the defective or missing gene by that of the therapeutic gene. Researchers have developed different types of vectors and techniques which facilitate gene transfer. For further information, click on gene therapy.

> Replacing deficient or missing cells by healthy cells – that’s the challenge of cell therapy. New leads are emerging in cell therapy, particularly through stem cells. As they are able to change into different types of cells, stem cells offer promising therapeutic prospects for the reconstruction of a damaged organ or tissue.
Therefore, the AFM has considerably reinforced its activities in the field of stem cells. For further information, click on cell therapy and stem cells.

> New leads are opening up which could have applications for many rare diseases. In particular, they allow direct intervention on the gene, or the “message” which is transmitted by the gene to the cell. For further information, click on exon skipping and new leads.
To know more about clinical trials.
To know more about neuromuscular diseases.