{"id":122929,"date":"2026-09-05T15:19:32","date_gmt":"2026-09-05T09:49:32","guid":{"rendered":"https:\/\/vajiramandravi.com\/current-affairs\/?p=122929"},"modified":"2026-09-05T15:28:01","modified_gmt":"2026-09-05T09:58:01","slug":"alexander-disease","status":"publish","type":"post","link":"https:\/\/vajiramandravi.com\/current-affairs\/alexander-disease\/","title":{"rendered":"Alexander Disease"},"content":{"rendered":"<h2><b>Alexander Disease Latest News<\/b><\/h2>\n<p style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Ionis Pharmaceuticals recently said it would price its newly approved therapy to treat Alexander disease at $285,000 per dose, a day after the U.S. FDA cleared the injectable \u200bdrug, making it the first approved treatment for the genetic \u200bdisorder in adults and children. <\/span><\/p>\n<h2><b>About Alexander Disease<\/b><\/h2>\n<ul>\n<li style=\"text-align: justify;\"><span style=\"font-weight: 400;\">It is a <\/span><b>rare genetic disorder<\/b><span style=\"font-weight: 400;\"> that <\/span><b>progressively damages<\/b><span style=\"font-weight: 400;\"> the <\/span><b>nervous system.\u00a0<\/b><\/li>\n<li style=\"text-align: justify;\"><span style=\"font-weight: 400;\">It is a <\/span><b>type of leukodystrophy,<\/b><span style=\"font-weight: 400;\"> a group of <\/span><b>conditions <\/b><span style=\"font-weight: 400;\">that<\/span><b> affect the white matter of the brain.\u00a0<\/b><\/li>\n<li style=\"text-align: justify;\"><span style=\"font-weight: 400;\">These diseases <\/span><b>damage the myelin sheath<\/b><span style=\"font-weight: 400;\">, which surrounds and <\/span><b>protects the nerve cells<\/b><span style=\"font-weight: 400;\"> in the brain and spinal cord and <\/span><b>speeds transmission of messages between cell<\/b><span style=\"font-weight: 400;\">s.\u00a0<\/span><\/li>\n<li style=\"text-align: justify;\"><b>In Alexander disease<\/b><span style=\"font-weight: 400;\">, the<\/span><b> myelin <\/b><span style=\"font-weight: 400;\">insulating the nerve fibers in the brain <\/span><b>deteriorates over time<\/b><span style=\"font-weight: 400;\">, and <\/span><b>abnormal clumps of protein<\/b><span style=\"font-weight: 400;\">, called <\/span><b>Rosenthal fibers, accumulate<\/b><span style=\"font-weight: 400;\"> in the brain.\u00a0<\/span><\/li>\n<li style=\"text-align: justify;\"><span style=\"font-weight: 400;\">This causes the<\/span><b> nervous system to stop working properly.\u00a0<\/b><\/li>\n<\/ul>\n<h2><b>Alexander Disease <\/b><b>Cause<\/b><\/h2>\n<ul>\n<li><span style=\"font-weight: 400;\">Most cases of Alexander disease are caused by a <\/span><b>mutation in the GFAP gene, <\/b><span style=\"font-weight: 400;\">which directs the body\u2019s production of a<\/span><b> glial fibrillary acidic protein (GFAP).\u00a0<\/b><\/li>\n<li><span style=\"font-weight: 400;\">At normal levels, <\/span><b>GFAP supports<\/b><span style=\"font-weight: 400;\"> the <\/span><b>brain\u2019s white matter (the myelin sheath).\u00a0<\/b><\/li>\n<li><span style=\"font-weight: 400;\">The <\/span><b>mutation <\/b><span style=\"font-weight: 400;\">of this gene in Alexander disease causes this <\/span><b>protein to accumulate.\u00a0<\/b><\/li>\n<li><span style=\"font-weight: 400;\">Instead of helping maintain the brain\u2019s white matter, the <\/span><b>extra GFAP <\/b><span style=\"font-weight: 400;\">does the opposite, <\/span><b>killing other cells and damaging the myelin.<\/b><\/li>\n<li><span style=\"font-weight: 400;\">In <\/span><b>most cases, <\/b><span style=\"font-weight: 400;\">the gene mutation associated with Alexander disease is<\/span><b> not inherited from a parent.\u00a0<\/b><\/li>\n<li><span style=\"font-weight: 400;\">It is simply a <\/span><b>random mutation <\/b><span style=\"font-weight: 400;\">and is new in the person who develops the syndrome.<\/span><\/li>\n<li><span style=\"font-weight: 400;\">In <\/span><b>some cases, <\/b><span style=\"font-weight: 400;\">Alexander disease is <\/span><b>inherited from<\/b><span style=\"font-weight: 400;\"> a similarly affected parent.\u00a0<\/span><\/li>\n<\/ul>\n<h2><b>Alexander Disease <\/b><b>Symptoms<\/b><\/h2>\n<ul>\n<li style=\"text-align: justify;\"><b>Most cases<\/b><span style=\"font-weight: 400;\"> of Alexander disease begin <\/span><b>before age 2 <\/b><span style=\"font-weight: 400;\">and are described as the infantile form.\u00a0<\/span>\n<ul>\n<li style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Signs and <\/span><b>symptoms <\/b><span style=\"font-weight: 400;\">of the infantile form typically include an <\/span><b>enlarged brain <\/b><span style=\"font-weight: 400;\">and <\/span><b>head size<\/b><span style=\"font-weight: 400;\"> (megalencephaly), <\/span><b>seizures<\/b><span style=\"font-weight: 400;\">, stiffness in the arms and\/or legs (spasticity),<\/span><b> intellectual disability,<\/b><span style=\"font-weight: 400;\"> and developmental delay.\u00a0<\/span><\/li>\n<\/ul>\n<\/li>\n<li style=\"text-align: justify;\"><b>Less frequently<\/b><span style=\"font-weight: 400;\">, onset occurs later in <\/span><b>childhood <\/b><span style=\"font-weight: 400;\">(the juvenile form) or in <\/span><b>adulthood<\/b><span style=\"font-weight: 400;\">.\u00a0<\/span>\n<ul>\n<li style=\"text-align: justify;\"><span style=\"font-weight: 400;\">Common problems in juvenile and adult forms of Alexander disease include<\/span><b> speech abnormalities, swallowing difficulties,<\/b> <b>seizures<\/b><span style=\"font-weight: 400;\">, and poor coordination (ataxia).\u00a0<\/span><\/li>\n<\/ul>\n<\/li>\n<li style=\"text-align: justify;\"><b>Rarely<\/b><span style=\"font-weight: 400;\">, a neonatal form of Alexander disease occurs within the <\/span><b>first month of life <\/b><span style=\"font-weight: 400;\">and is associated with <\/span><b>severe intellectual disability and developmental delay,<\/b><span style=\"font-weight: 400;\"> a <\/span><b>buildup of fluid in the brain (hydrocephalus),<\/b><span style=\"font-weight: 400;\"> and seizures.\u00a0<\/span><\/li>\n<\/ul>\n<h2><b>Alexander Disease <\/b><b>Treatment<\/b><\/h2>\n<ul>\n<li style=\"text-align: justify;\"><span style=\"font-weight: 400;\">There is <\/span><b>no complete cure<\/b><span style=\"font-weight: 400;\"> for Alexander disease, but the U.S. FDA <\/span><b>recently approved Zanvastro (zilganersen)<\/b><span style=\"font-weight: 400;\">, which is the <\/span><b>first disease-modifying therapy <\/b><span style=\"font-weight: 400;\">that directly targets the underlying protein buildup driving the condition.<\/span><\/li>\n<li style=\"text-align: justify;\"><span style=\"font-weight: 400;\">The disease is <\/span><b>often fatal. <\/b><\/li>\n<\/ul>\n<p><b>News: <\/b><a href=\"https:\/\/www.reuters.com\/legal\/litigation\/us-fda-approves-ionis-pharmas-therapy-treat-rare-brain-disorder-2026-09-03\/\" target=\"_blank\" rel=\"nofollow noopener\"><b>REUT<\/b><\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Alexander Disease is a rare genetic disorder that progressively damages the nervous system. Read more about Alexander Disease, Cause, Symptoms, Treatment, Latest News.<\/p>\n","protected":false},"author":23,"featured_media":122993,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[1],"tags":[10040,21,22,23],"class_list":["post-122929","post","type-post","status-publish","format-standard","has-post-thumbnail","category-upsc-prelims-current-affairs","tag-alexander-disease","tag-prelims-pointers","tag-upsc-current-affairs","tag-upsc-prelims-current-affairs","no-featured-image-padding"],"acf":[],"_links":{"self":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/posts\/122929","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/users\/23"}],"replies":[{"embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/comments?post=122929"}],"version-history":[{"count":4,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/posts\/122929\/revisions"}],"predecessor-version":[{"id":123026,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/posts\/122929\/revisions\/123026"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/media\/122993"}],"wp:attachment":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/media?parent=122929"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/categories?post=122929"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/tags?post=122929"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}