{"id":352986,"date":"2025-12-30T16:20:02","date_gmt":"2025-12-30T10:50:02","guid":{"rendered":"https:\/\/forumias.com\/blog\/?p=352986"},"modified":"2025-12-30T16:20:02","modified_gmt":"2025-12-30T10:50:02","slug":"itvisma-gene-therapy","status":"publish","type":"post","link":"https:\/\/forumias.com\/blog\/itvisma-gene-therapy\/","title":{"rendered":"ITVISMA Gene Therapy"},"content":{"rendered":"<p><strong>News: <\/strong>Abu Dhabi\u2019s Sheikh Khalifa Medical City became the first hospital to administer ITVISMA after UAE regulatory approval in November 2025.<\/p>\n<h2>About ITVISMA Gene Therapy<\/h2>\n<figure id=\"attachment_352987\" aria-describedby=\"caption-attachment-352987\" style=\"width: 308px\" class=\"wp-caption aligncenter\"><img data-recalc-dims=\"1\" loading=\"lazy\" decoding=\"async\" class=\"wp-image-352987 \" src=\"https:\/\/i0.wp.com\/forumias.com\/blog\/wp-content\/uploads\/2025\/12\/Source-U.A.E.-Govt.jpg?resize=308%2C340&#038;ssl=1\" alt=\"ITVISMA Gene Therapy\" width=\"308\" height=\"340\" srcset=\"https:\/\/i0.wp.com\/forumias.com\/blog\/wp-content\/uploads\/2025\/12\/Source-U.A.E.-Govt.jpg?resize=272%2C300&amp;ssl=1 272w, https:\/\/i0.wp.com\/forumias.com\/blog\/wp-content\/uploads\/2025\/12\/Source-U.A.E.-Govt.jpg?w=656&amp;ssl=1 656w\" sizes=\"auto, (max-width: 308px) 100vw, 308px\" \/><figcaption id=\"caption-attachment-352987\" class=\"wp-caption-text\">Source &#8211; U.A.E. Govt.<\/figcaption><\/figure>\n<ul>\n<li><strong>&#8220;Itvisma&#8221;<\/strong> is a brand name for the gene therapy drug <strong>onasemnogene abeparvovec-brve.<\/strong><\/li>\n<li>It \u00a0is a <strong>one-time gene therapy<\/strong> approved in late 2025 for the treatment of <strong>spinal muscular atrophy (SMA)<\/strong> in adults and children aged 2 years and older.<\/li>\n<li>The therapy <strong>addresses the genetic cause of the disease rather<\/strong> than only managing symptoms.<\/li>\n<li><strong>Developed by:<\/strong> ITVISMA was developed by <strong>Novartis<\/strong>.<\/li>\n<li><strong>Aim<\/strong>: The treatment aims to <strong>improve motor function and reduce long-term treatment dependence.<\/strong><\/li>\n<li><strong>Mechanism<\/strong>\n<ul>\n<li><strong>SMN1 Gene:<\/strong> This is the <strong>gene<\/strong> that is <strong>mutated or missing in people<\/strong> with <strong>spinal muscular atrophy (SMA)<\/strong>, the condition Itvisma treats.<\/li>\n<li>The <strong>gene&#8217;s role<\/strong> is to <strong>produce the essential SMN protein<\/strong> needed for <strong>motor neuron function, muscle movement, breathing, and swallowing.<\/strong><\/li>\n<li><strong>ITVISMA&#8217;s role:<\/strong> It is an <strong>adeno-associated virus (AAV) vector-based therapy<\/strong> designed to <strong>deliver a new, working copy of the SMN1 gene directly to the motor neurons<\/strong> in the <strong>spinal cord<\/strong> via a one-time intrathecal injection.<\/li>\n<li><strong>Result:<\/strong> The <strong>new gene enables<\/strong> continuous and sustained production of the SMN protein, <strong>addressing the genetic root cause<\/strong> of SMA to improve or stabilize motor function.<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<ul>\n<li><strong>Risk of Therapy<\/strong>\n<ul>\n<li><strong>Common Side Effects:<\/strong> Common side effects include fever, vomiting, headache, and upper respiratory tract infections.<\/li>\n<li><strong>Serious Risks:<\/strong> Serious risks include liver toxicity, heart-related effects, and reduced platelet counts.<\/li>\n<\/ul>\n<\/li>\n<li><strong>Significance :<\/strong> ITVISMA can deliver <strong>sustained motor-function improvement with a single dose<\/strong>, reduce lifelong treatment dependence, and expand access beyond infant-only therapies.<\/li>\n<\/ul>\n","protected":false},"excerpt":{"rendered":"<p>News: Abu Dhabi\u2019s Sheikh Khalifa Medical City became the first hospital to administer ITVISMA after UAE regulatory approval in November 2025. About ITVISMA Gene Therapy &#8220;Itvisma&#8221; is a brand name for the gene therapy drug onasemnogene abeparvovec-brve. It \u00a0is a one-time gene therapy approved in late 2025 for the treatment of spinal muscular atrophy (SMA)&hellip; <a class=\"more-link\" href=\"https:\/\/forumias.com\/blog\/itvisma-gene-therapy\/\">Continue reading <span class=\"screen-reader-text\">ITVISMA Gene Therapy<\/span><\/a><\/p>\n","protected":false},"author":10393,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"jetpack_post_was_ever_published":false,"footnotes":""},"categories":[1566,1738,12039],"tags":[11872],"class_list":["post-352986","post","type-post","status-publish","format-standard","hentry","category-daily-factly-articles","category-science-and-technology-daily-factly-articles","category-knolls","tag-9pm-daily-factly","entry"],"jetpack_featured_media_url":"","views":"","jetpack_sharing_enabled":true,"_links":{"self":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts\/352986","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/users\/10393"}],"replies":[{"embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/comments?post=352986"}],"version-history":[{"count":0,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts\/352986\/revisions"}],"wp:attachment":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/media?parent=352986"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/categories?post=352986"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/tags?post=352986"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}