Find here all the latest news on AFM-Telethon and rare diseases.
Because muscles are a major public health issue, AFM-Telethon and the Institute of Myology, an international center of expertise on muscles and their diseases, are carrying out a project for a Myology Foundation. In addition, to raise awareness on myology among the public, they are launching two events dedicated to this public health issue in June 2023: the Muscle Conferences and Muscle Week.
Their determination and expertise are at the origin of great scientific advances and major victories. Thanks to the researchers in the Telethon laboratories, children suffering from formerly incurable diseases have been saved.
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), has launched a pivotal clinical trial in Europe for treatment of Crigler-Najjar syndrome, a life threatening liver disease, in France, Italy and the Netherlands.
The AFM-Telethon sends you its best wishes for the New Year and wishes you a year 2023 full of color and joy!
"Advances in Charcot-Marie-Tooth (CMT) disease" is a new document, published by the French Muscular Dystrophy Association (AFM-Téléthon), which can be read and/or downloaded here.
During four days, from 12 to 15th of September, researchers, clinicians and myology experts have attended the seventh International Congress of Myology, organized by AFM-Téléthon, in Nice, France. A good opportunity to exchange and share knowledge about myology and innovative therapies. Let’s have a look back at Myology 2022 with our researchers’ video interviews.
From September 12 to 17th, AFM-Telethon will hold two successive scientific congresses gathering close to 1000 international experts on myology and mitochondrial medicine at the Nice Acropolis Convention Centre. Registrations are open!
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
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.
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.
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.
On February 28, International Rare Disease Day will put the spotlight on the rare diseases community throughout the world. At this occasion, AFM-Telethon revisits thirty years of pioneering research and innovation carried out by its laboratory, Genethon, which has developed a high-level expertise in researching and developing preclinical and clinical gene therapy treatments for rare diseases.
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.