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In an article published in July 2023, an Italian researcher traces the birth and development of myology. This medical science of muscle and its diseases has developed over time, not only thanks to the advent of tools and technologies that improve our knowledge of muscle, but also thanks to the international collaborations that have marked its history.
Results published in The New England Journal of Medicine demonstrate the safety and tolerance for gene therapy in Crigler-Najjar syndrome as well as its efficacy at the highest dose. This rare liver disease is characterized by a toxic build-up of bilirubin in the body, which can cause significant neurological damage and become fatal.
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!
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.
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.
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.
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.
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!
The Téléthon 30-hour-long broadcast ended with donations totaling 78 051 091 euros. From Guebwiller to Cassis, Dijon, Lorient and Cap-Ferret, millions of people came together again for the Téléthon, this unique celebration with its thousands of events. A vital support for the families and researchers, “these saving lives maniacs” as Kev Adams called them. But while researchers’ victories multiply, all week-end long patients and their families reminded everyone of the urgency of finding new treatments.
"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.
You live abroad and want to play a part in the Telethon? Join the Telethon for French people abroad and help us break records of mobilization around the world!