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Find here all the latest news on AFM-Telethon and rare diseases.
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Happy new year!
The AFM-Téléthon sends you its best wishes for the New Year and hopes that 2024 will be full of scientific breakthroughs, small and large joys, and shared victories!
I-Stem, coordinator of an innovative research consortium
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!
Myotubular muscular dystrophy: trial results show efficacy on respiratory and motor function, but challenges remain
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.
Limb-Girdle Muscular Dystrophy Type 2I/R9: first gene therapy results
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.
Human induced pluripotent stem cells, a valuable tool for better understanding and treating neuromuscular diseases
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.
LGMD Awarness Day: Stronger Together!
The 30th of September is Limb-Girdle Muscular Dystrophy Awareness Day. At this occasion, the LGMD Interest Group of the AFM-Téléthon is planning an information day about these diseases in Evry.
World Duchenne Awareness Day: everyone mobilized !
The 10th World Duchenne Awareness Day, on September 7, is a call to action to break down the physical, social and healthcare barriers faced by the 250,000 people around the world affected by this disease. It's also an opportunity to take stock of advances in research into this disease, which is emblematic of our Association.
The history of myology in Europe
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.
Crigler-Najjar: gene therapy shows its effectiveness
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.
The new Cure through Innovation booklet is out!
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!
Genother Biocluster: Accelerating the development of gene therapy drugs in France
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.
A new president for the Scientific Advisory Board
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.
Licence agreement for the development of a treatment for Amyotrophic Lateral Sclerosis (ALS)
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.
Protagonists of a medical revolution, researchers testify
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.