Key figures: €60.6 million to cure rare diseases

In 2015, thanks to Telethon donations, the French Muscular Dystrophy Association (AFM-Téléthon) invested € 63.8 million in rare disease research. Every year, it devotes about two-thirds of its missions budget to this prime concern.

Breakdown of 2015 funding per research centre

Généthon: €25.1 M
Institute of Myology: € 8.6 M
I-Stem: €4.7 M
Atlantic Gene Therapies: € 2.4 M

Breakdown of 2015 funding per type of research

Research and development: €7.1 M (12.9%)
Basic research: €8.2 M (12.9%)
Gene therapy: €31.6 M (52.2%)
Cell therapy: €2M (8.3%)
Pharmacology: €5.3 M (8.3%)
Cross-cutting projects: €11.1 M (17.4%)
Valorisation: €1.2 M (1.8%)

20 Mar 2018

The 31th Telethon edition reaches a final collection of 89,189,384 euros

On 8th and 9th December 2017, the Telethon enthralled the public throughout France.

14 Mar 2018

Spinal muscular atrophy : AveXis enters into licensing agreement with Genethon

AveXis, Inc. and Genethon today announced they have entered into an exclusive, worldwide license agreement for in vivo gene therapy delivery of AAV9 vector into the central nervous system (CNS) for the treatment of spinal muscular atrophy (SMA).

13 Feb 2018

Gene therapy: Généthon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler-Najjar Syndrome

Illustration for news: Gene therapy: Généthon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler-Najjar Syndrome

Généthon, a laboratory created by AFM-Téléthon, starts a European phase I/II clinical trial to test a treatment for Crigler-Najjar Syndrome, a rare liver disease.

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