- 9 September 2019 A gene therapy based clinical trial for Fanconi anemia patients offers its first successful results
- 26 June 2019 First in vivo proof-of-concept in Steinert’s myotonic dystrophy, a neuromuscular disease
- 25 May 2019 Genethon welcomes the first FDA-approved gene therapy for a neuromuscular disease and emphasizes its decisive role in this historic step
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.