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!
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.
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.
This booklet features AFM-Telethon’s main missions, actions and key figures.
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.