The Telethon is back! This Saturday December 7 and Sunday December 8, 2018, the AFM-Telethon association is mobilizing to collect donations in favor of medical research against genetic and neuromuscular diseases.
Founded in 1958, the French Association against Myopathies (AFM) has supported therapies based on knowledge of genes for more than 20 years, in particular gene and cell therapy. And progress is concrete: in 2018, overcoming the disease seems (finally) possible!
For example, in September 2017, a child with myotubular myopathy (a genetic disease that affects 1 in 50,000 babies and is characterized by extreme muscle weakness and severe respiratory failure) was treated in the United States with a product gene therapy initially designed and developed by Généthon, the Telethon laboratory. Three months after the injection, the child is sitting alone and is able to pick up objects.
Beating genetic and neuromuscular diseases is finally possible
“Very encouraging results” have also been obtained in children with spinal muscular atrophy (a rare genetic disease that affects motor neurons, leading to progressive muscle atrophy) via gene therapy that Genethon helped to develop. Thanks to a single injection, 15 babies were treated: by the age of 24 months, almost all of them are able to sit up unaided, and a third of them can stand.
Finally, in November 2017, a 7-year-old boy with junctional epidermolysis bullosa (a rare genetic disease characterized by the appearance of bubbles all over the body) was saved thanks to a world first: a genetically engineered skin graft. modified on 80% of the body. A medical feat partly enabled by the Telethon!
To donate, call 3637. To donate online, go to telethon.fr.
Read also :
Myopathy: “It hurts to know that animals are going to suffer just for me”.
Telethon: thanks to donations Léo takes part in a clinical trial and walks!
A 5-year-old child saved thanks to Telethon donations
