- 12 February 2018 Genethon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler-Najjar Syndrome
- 12 January 2018 First promising results towards the treatment of myotubular myopathy
- 15 December 2017 A gene therapy alliance in X-linked chronic granulomatous disease with Orchard Therapeutics
Created in 1990 by AFM-TELETHON, Genethon is fully dedicated to the design and development of gene therapy treatments for rare diseases. Its objective is to provide these innovative treatments to patients affected with rare disorders.
Genethon is developing therapies for rare neuromuscular diseases, immune system or blood disorders, eye disorders and liver diseases.