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 →
The 3rd MLD Alliance international congress on newborn screening for metachromatic leukodystrophy (MLD) was held in Florence on November 3rd and 4th 2022. It brought together some sixty clinicians, scientists, industry and patient association representatives. The 1st day, some specific workshops were organized. Scientists shared information about the ongoing newbornRead More →
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 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 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 →
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 →
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 →
