Do a search on the AFM-Telethon website.
From genome maps to the discovery of the genes responsible for diseases, an entire sector of world medicine has made a spectacular leap forward, thanks to the determination of AFM-Telethon families, and the momentum of the Telethon.
Read more about AFM-Telethon’s social missions, main activities, and key figures in this new edition of Cure through Innovation.
Through the collective mobilization of donors, volunteers, families, and partners, all united in solidarity to fight against rare diseases, the final result of the 2021 Telethon amounts to €85.933.166. Thank you!
From September 12 to 17, 2022, AFM-Telethon will hold two international scientific congresses at the Nice Acropolis Convention Centre. You can submit your abstracts until April 30, 2022
It is a positive commitment, but France must set an example by perpetuating and amplifying a truly ambitious national policy.
Myology Institute's team has developed and tested a new approach to gene therapy, a so-called “decoy” approach, for Steinert’s disease or myotonic dystrophy type 1.
Genethon announced that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.
On 16 December 2021, the UN adopted the first-ever UN Resolution on “Addressing the Challenges of Persons Living with a Rare Disease and their Families.” A huge recognition for families with rare diseases.
The 2021 Telethon ended with donations totaling 73 622 019 euros. An amount reflecting the generosity of the general public, the energy felt during thousands of Telethon events throughout France and abroad, and the strength of families’ fight and researchers’ determination. This unique combination is the cause for a true revolution in medicine that saves lives and opens up new prospects for treatment.
Families, researchers, volunteers, partners… all are ready to organize the 35th Telethon! A crucial event for patients and their family, and for the whole of medicine.
This booklet features AFM-Telethon’s main missions, actions and key figures.
« Advances in Steinert’s disease » and « Advances in myotonic dystrophy type 2 » are two new documents, published by the French Muscular Dystrophy Association (AFM-Téléthon), which can be read and/or downloaded here.
Preliminary results from the European gene therapy trial for Crigler-Najjar syndrome, conducted by Généthon in collaboration with European network CureCN, were presented at the EASL (European Association for the Study of the Liver) annual International Liver Congress on June 26. Based on initial observations, the drug candidate is well tolerated and the first therapeutic effects have been demonstrated, to be confirmed as the trial continues.
Last December 4th and 5th, the 2020 Telethon took place in a difficult context due to the COVID19 crisis with donations totalling nearly 58.3 million euros by the end of the TV broadcast. A few months later, we are extremely proud to share the exceptional final result of our 2020 fundraising campaign: 77 298 024 euros. Thank you so much!
A first participant was dosed at I-Motion, the pediatric clinical trial platform for neuromuscular diseases located at Trousseau hospital in Paris, as part of the gene therapy trial in Duchenne muscular dystrophy (DMD) conducted by Genethon.