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Developing innovative therapies

Correcting a defective gene, inserting a gene-drug, grafting cells capable of regenerating healthy tissue: AFM-Téléthon paves the way for the medicine of the future and supports about 37 clinical trials.

Illustration for category: Gene surgery : repairing the gene

Gene surgery : repairing the gene

Gene surgery involves repairing the gene itself. Exon skipping, stop codon read-through, meganucleases: these techniques are less than ten years old but already undergoing human trials!
Illustration for category: Cell therapy : replacing the deficient cells

Cell therapy : replacing the deficient cells

Cell therapy takes up the challenge of replacing deficient or absent cells with healthy cells. It succeeds mainly through the contribution of specific cells: stem cells and iPS. From cardiac insufficiency to neuromuscular disease via cancer or nervous system pathologies, there are numerous applications.
Illustration for category: Drugs: a traditional pathway

Drugs: a traditional pathway

New molecules are constantly being discovered and existing drugs have not yet reached their full potential. AFM-Téléthon supports research into new molecules as well as existing pharmacological treatments.
Illustration for category: Ongoing trials and projects

Ongoing trials and projects

Every year AFM-Téléthon supports clinical trials designed to evaluate the efficacy of innovative treatments. Using Telethon donations, it supports 37 clinical trials for 27 different diseases.
Illustration for category: Gene therapy, a booming therapeutic sector

Gene therapy, a booming therapeutic sector

Gene therapy offers the possibility of replacing the function of the defective or missing gene with that of a therapeutic gene inserted into the cell. The first diseases which benefited from gene therapy were rare genetic diseases in which the gene responsible had been identified, severe immune deficiencies.