The patients’ voice has been heard!
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 →...
Like 11 European countries, the United States approves the Libmeldy
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 →...
International Rare Disease Day: the policy of the 1st child who saves the 2nd is intolerable.
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 →...
Scientific Committee of ELA International: a face to face much awaited
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 →...
Mets tes baskets et bats la maladie !
Avec Tony Estanguet, Zinédine Zidane, Florent Pagny et tous les ambassadeurs d’ELA, relevons cet incroyable défi : être 1 million sur la ligne de départ et faire un 1 milliard de pas pour aider les enfants malades. Prêts à mettre vos baskets ? “Mets tes baskets” invite chaque année lesRead More →...
Sign the petition to save children from being denied treatment for commercial reasons
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 Family Weekend Switzerland 2023
From August 4 to 6, 2023, the ELA Suisse team welcomed the association’s families to the Hôtel Cailler in Charmey (canton of Fribourg). On Saturday morning, the children had the pleasure of trying out different games in a workshop led by Béatrice Hess (honorary member of ELA). During this activity,Read More →...
For the first time, a boy receives a commercially available gene therapy for cerebral X-linked adrenoleukodystrophy (ALD) in the United States
On March 16th, 2023, after thirty years of research started in France with the support of ELA, the first boy in the world received the FDA approved gene therapy for the cerebral form of adrenoleukodystrophy. Following a positive newborn screening, 6-year-olds Conner Hess from New York was followed closely toRead More →...
