Very happy to collaborate with ELA Spain in the VI Charity Rake, where the importance of including leukodystrophies in neonatal tests for early diagnosis was highlighted. This rare disease affects one in every 7,500 people, mostly children. The president of ELA Spain, Carmen Sever, emphasized the urgency of detecting these disorders at time, since gene therapy treatments have a narrow therapeutic window.
Leukodystrophies are devastating, destroying or preventing the proper formation of myelin, which affects communication between the brain and the body. The fight against ALS has made significant progress in treatments and faster diagnosis. The Government delegate in Castilla y León, Virginia Barcones, urged progress in research and highlighted the importance of early detection. Several authorities supported the event, which raises funds to help affected families and finance research.
PDF with data from the VI Charity Rake:
