Find here all the latest news on AFM-Telethon and rare diseases.
On June 20, World FSHD Day reminds us of the importance of raising awareness and supporting research into facioscapulohumeral muscular dystrophy. Committed to the fight against FSHD, the AFM-Téléthon is making available a series of publications to enlight the advances in research by it, but also by many other neuromuscular diseases.
The initial results of Genethon's gene therapy trial for Duchenne muscular dystrophy were presented on Tuesday 23 April at the Myology 2024 congress in Paris.
Finding treatments for neuromuscular diseases by combining artificial intelligence, stem cells and pharmacological screening is the aim of the new DREAMS research consortium, coordinated by I-Stem. An unprecedented project!
The 2024 edition of the International Consortium on Myotonic Dystrophy (IDMC-14) will take place in Nijmegen from 9 to 13 April 2024. This is an opportunity to learn, share and network thanks to the presence of scientists, clinicians, patients and professionals from the pharmaceutical industry.
A gene therapy developed at Genethon by Ana Buj Bello's team is proving effective in a rare and very severe muscle disease: myotubular myopathy. Review of the results published online in The Lancet Neurology on November 15.
The 8th edition of international congress dedicated to neuromuscular science, MYOLOGY 2024, will take place in Paris from 22 to 25 April 2024. Registration and call for abstract are now open : don't miss an opportunity to showcase your latest study to an international community of scientists and healthcare professionals. The deadline for abstract submission has been extended until November 30, 2023 - 11:59 pm.
The first clinical results of the gene therapy for the treatment of limb-girdle muscular dystrophy type 2I/R9 show good preliminary efficacy and biological tolerability. These first results have been presented during oral communications during the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) and will be presented in the 2023 International Limb-Girdle Muscular Dystrophy Conference in Washington DC.
A review published by two researchers at I-Stem, one of the AFM-Téléthon laboratories, takes stock of human pluripotent stem cells (hiPS) and confirms their main advantages in neuromuscular diseases: improving understanding and identifying new treatments.
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.
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.
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!