Find here all the latest news on AFM-Telethon and rare diseases.
Characterized by fatigue and muscle weakness that fluctuate over time, autoimmune myasthenia gravis is the subject of the very first European Myasthenia Gravis Day, on 2 June 2023. The aim is to give as many people as possible an understanding of the impact of this unpredictable and often invisible disease, and to standardize the way it is treated across Europe.
The Genother biocluster, dedicated to accelerating the development of gene therapy drugs, of which Genethon is one of the founders, is one of the 5 winners of the France 2030 call for expressions of interest. This labelling, announced on 16 May by the French President, will strengthen France's leadership in the field of gene therapy, one of the most dynamic areas of biomedical research.
Cure through Innovation is a booklet that sums up the main actions and missions of the AFM-Téléthon association, as well as the key figures for 2022. The July 2023 edition has just been published!
Thank you to everyone: donors, volunteers mobilized throughout France and abroad, families, partners, personalities! Thanks to your mobilization on December 2 and 3, the Telethon 2022 raised 90,839,067 euros! This tremendous result - the best since 2016 - shows how much the French people support the families’ fight against rare diseases.
ILTOO Pharma and the European consortium MIROCALS have just announced the signing of a license agreement for the development of a low-dose Interleukin 2 as a potential treatment for amyotrophic lateral sclerosis (ALS). The AFM-Telethon helped finance this clinical study, which shows an improvement in patient survival.
On April 12, Prof. Judith Melki succeeded Odile Boespflug-Tanguy as president of the AFM-Telethon’s Scientific Advisory Board. Professor Emeritus of medical genetics, Judith Melki is at the origin of the discovery of the SMN gene, responsible for spinal muscular atrophy.
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.
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.
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.
At the end of the television broadcast, the counter of the 2020 Telethon shows 58 290 120 euros. Despite the health crisis, which led to the cancellation of a very large number of events in towns and villages, the French people responded positively to this extraordinary Telethon: the one presenting the greatest victories of its history. Behind these victories, there are faces, first names, children, parents, changed lives, regained motions, smiles and laughter, and life who wins.
Généthon, a unique scientific and human adventure, turns 30 this year. Created by a patient organization and funded by the Téléthon, this laboratory has put France at the forefront of genome exploration and of the gene therapy revolution.
What is AFM-Telethon? What are its missions? What were the highlights of 2019? The answer is in the Cure through Innovation booklet.
Enabling early detection and diagnosis of the disease is the aim of the new European Alliance for Newborn Screening in Spinal Muscular Atrophy (SMA NBS Alliance), launched on August 31st by SMA Europe, of which AFM-Telethon is a member.
Four types of myositis that consider all the clinical criteria of patients have been defined. A new classification that paves the way for reliable diagnosis and personalised treatments for patients.
« 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.