{"id":159072,"date":"2023-02-26T23:22:22","date_gmt":"2023-02-27T05:22:22","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2023\/02\/new-hope-for-treatment-of-rare-metabolic-brain-disease"},"modified":"2023-02-26T23:22:22","modified_gmt":"2023-02-27T05:22:22","slug":"new-hope-for-treatment-of-rare-metabolic-brain-disease","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2023\/02\/new-hope-for-treatment-of-rare-metabolic-brain-disease","title":{"rendered":"New Hope for Treatment of Rare Metabolic Brain Disease"},"content":{"rendered":"<p><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/new-hope-for-treatment-of-rare-metabolic-brain-disease2.jpg\"><\/a><\/p>\n<p>X-ALD is the most prevalent of the approximately 50 rare diseases that affect the white matter of the brain, referred to as leukodystrophies. The genetic damage in X-ALD is due to a defect in the X chromosome. Men who are affected by X-ALD experience a progressive deterioration of their mobility, balance, and sensory abilities, leading to issues such as incontinence and sexual dysfunction.<\/p>\n<p>Although X-ALD is inherited through the X chromosome, female carriers can also experience symptoms of the disease. Approximately 30% of male children and 60% of adult men develop encephalitis, which is a fatal form of the disease that leads to death within two to three years. X-ALD affects roughly one in every 20,000 births globally.<\/p>\n<p>Now, for the first time, scientists from all relevant leukodystrophy centers in Europe and the US have jointly succeeded in obtaining controlled trial data for X-linked adrenoleukodystrophy. Of the 116 patients, 77 received the drug leriglitazone and 39 a placebo. The drug had already shown in preclinical studies that it can prevent neurodegeneration and offer protection against the life-threatening inflammation of the brain.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>X-ALD is the most prevalent of the approximately 50 rare diseases that affect the white matter of the brain, referred to as leukodystrophies. The genetic damage in X-ALD is due to a defect in the X chromosome. Men who are affected by X-ALD experience a progressive deterioration of their mobility, balance, and sensory abilities, leading [\u2026]<\/p>\n","protected":false},"author":511,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,412,47],"tags":[],"class_list":["post-159072","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-genetics","category-neuroscience"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/159072","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/511"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=159072"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/159072\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=159072"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=159072"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=159072"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}