Symposium virtuel ELA International 2022 – To review the symposium
ELA International organized a virtual symposium on Saturday September 24, 2022 Therapeutic innovation: what ethical rules and what access? This event is a continuation of a first important event organized in last November on Clinical trials and Ethics. It brought together medical experts, industrialists, health agencies and families to exploreRead More →
ELA Families/Researchers meeting 2022 : VIDEOS AVAILABLE
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 →
ALD connect meeting on Friday, July 22
ALD connect will be holding an Externally-Led Patient-Focused Drug Development meeting on Friday, July 22 to inform the Food and Drug Administration, about what it is like to live with ALD. The event will be open to the public online and free of charge. For more information:Read More →
Study on the quality of life of women with X-ALD: two new sites are open
Two new sites are open for the study on the quality of life of women with X-ALD on Leuconnect The study on the quality of life of women with X-ALD is now open in Spanish and Italian on Leuconnect. This study is an extension of the first study already availableRead More →
Publication of recommendations Guidelines for Clinical Trials in CACH Syndrome (VWM)
9 clinician-scientists, experts in CACH Syndrome, met in a consortium. They represent the countries of the International Council for Harmonization–Good Clinical Practice guidelines. CACH Syndrome is a genetic disease of the hypomyelinizing leukodystrophy family, with a progressive degradation of the white matter of the brain. There is no treatment atRead More →
Virtual SYMPOSIUM ELA International « CLINICAL TRIALS AND ETHICS » Saturday November 27th – 3:00 PM – 6:00 PM (CET)
Virtual SYMPOSIUM ELA International « CLINICAL TRIALS AND ETHICS » Saturday November 27th, 2021 ELA international organized an innovative symposium aiming to reflect how patients and their families can be involved in the various research projects that concern them, and to define the ethical rules that will guide ELA InternationalRead More →
2021 : A major advancement in the treatment of adrenoleukodystrophy
Thanks to ELA massive investment in research on cerebral adrenoleukodystrophy (CALD), the team of Pr. Patrick Aubourg and Dr. Nathalie Cartier published in 2009 the first results of a gene therapy trial on cerebral adrenoleukodystrophy conducted in France. This research was extended internationally by the Bluebird Bio laboratory which conductedRead More →
1 JUNE 2021: OPENING OF THE 1st CLINICAL TRIAL FOR CACH SYNDROME
The disease CACH (Childhood Ataxia with Central Nervous System Hypomyelination) is a rare genetic disease of the hypomyelinating leukodystrophy family, also known as leukoencephalopathy with vanishing white matter (VWM). It is characterised by a progressive degradation of the white matter in the brain, leading to various cognitive disorders and muscleRead More →
