ELA international is giving a voice to the parents of sick children who are speaking out against the planned discontinuation of a treatment currently under trial Scheduled discontinuation in 2024 In 2012, Takeda launched a clinical trial to evaluate the efficacy of an enzyme replacement therapy (TAK-611) in metachromatic leukodystrophyRead More →
A rare and devastating genetic disease Metachromatic leukodystrophy (MLD) is a rare genetic disease, occurring at a rate of 1 in 45,000 births. It can begin in childhood, adolescence, or adulthood, and leads to severe neurological dysfunction affecting motor skills and cognitive functions, and potentially to death. Libmeldy: an innovativeRead More →
ELA International warns of the importance of screening for leukodystrophies at birth. Diagnosed too late, Alice lost her speech and mobility in less than a month. As a result of this cataclysm, her little sister Coline was screened very early, even before the first symptoms of the disease appeared. That’sRead More →
This year, the autumn Scientific Committee meeting took place in a conference hotel in the center of Paris on Monday, October 23rd, for the first time in 3 years. Members of committee were happy to be gathered again for a studious day that allowed the evaluation of all the applicationsRead More →
Mathéo could have been cured with a gene therapy invented in France. He was denied treatment for commercial reasons. To prevent this story from happening again, a mother of ELA calls out the French Ministers of Health and Research, as well as the Secretary General for Investment-France 2030 through aRead More →
ELA international repeats the ELA Families/Researchers meeting virtually, an event open to the international community of patients affected by leukodystrophy. A simultaneous translation in 5 languages (German, English, Spanish, French, Italian) allowed everyone to follow the presentations live. The videos of the meeting are available. You can review it now:Read More →
ELA International, partner of NG4Leuko’s program led by Vivi Heine, professor of neuroscience at the University of Amsterdam, organized a review meeting in Paris which brought together 5 teams of European researchers involved in the project. After a convivial diner organized in a typical Parisian brasserie on Thursday, researchers hadRead More →
Following the symposium organized by ELA International in September 2022 on “Therapeutic Innovation: what rules and access?”, ELA International decided to set up a research ethics committee. This committee brings together 11 experts in ethics, law, human sciences, representatives of ELA families, and scientists, to promote a collegial and multidisciplinaryRead More →
Since its creation in 1992, ELA has forged special ties with Nathalie Cartier and has strongly supported her work. She participated in several events to support the actions of ELA and was a member of the Scientific Committee of ELA International from 2018 to 2022. At a time when geneRead More →
With the mobilization of the families, health professionals and ELA, and under the lead of the firm ARGO Santé, a study on the impact of cerebral adrenoleukodystrophy on the quality of life of patients and their families was conducted on Leuconnect. The platform allowed the expected recruitment and a rapidRead More →
