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Genethon, a pionner and a leader in the discovery and development of gene therapies for rare diseases, is a unique non-profit organization created by a patient association, the AFM-Telethon.

Genethon has multiple ongoing programs at clinical, preclinical and research stage for neuromuscular, blood, immune system, and liver diseases.

A first gene therapy drug, to which Genethon contributed, has obtained marketing for spinal muscular atrophy. (Zolgensma)

With 200+ scientists and professionals, Genethon is pursuing its mission to bring life-changing therapies to patients suffering from rare genetic diseases.

13 products resulting from Genethon’s research are in clinical trials for eye, liver, blood, immune system and muscle diseases. A further 6 products are in the preparation phase for clinical trials over the next five years.

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