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, 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 →
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 →
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 →
The United Leukodystrophy Foundation (ULF) in the United States, in partnership with ELA International, organizes the Unlock CTX virtual meeting focused on the leukodystrophy cerebrotendinous xanthomatosis CTX, under the US Food and Drug Administration’s (FDA) Patient-Focus Drug Development (PFDD) program. It will take place on September 14, 2021 in theRead More →
Every year, ELA organizes a meeting in Paris which brings families and researchers together affected by leukodystrophies. This event could not take place in 2020 due to the global pandemic. The health context having made it impossible to organize the Families/Researchers meeting in person, ELA has had to adapt toRead More →
Every week in Europe 20 to 40 children are born with a form of rare disease called leukodystrophy that attacks and destroys the central nervous system (brain and spinal cord) in children and adults. But research is improving thanks to a € 26 200 donation made in 2018 to theRead More →
Call for proposals ELA 2021 ELA International represents national ELA associations from different countries that contribute together to the financing of research on leukodystrophies. It is run by parents and /or patients affected by leukodystrophies. Accordingly, ELA International wishes to focus funding on programs devoted to the improvement of theRead More →
Study on the quality of life of women affected by X-linked adrenoleukodystrophy [ALD] A new study is available on Leuconnect! In partnership with ELA International and under the responsibility of Professor Wolfgang Koehler in Germany, the team from the University of Leipzig is opening a study on the quality ofRead More →
