{"id":2456,"date":"2024-11-27T13:55:49","date_gmt":"2024-11-27T12:55:49","guid":{"rendered":"https:\/\/rvh-synergie.org\/actualites\/la-fda-approuve-kebilidi-pour-le-deficit-en-decarboxylase-dacide-l-amine-aromatique\/"},"modified":"2024-11-27T13:55:50","modified_gmt":"2024-11-27T12:55:50","slug":"la-fda-approuve-kebilidi-pour-le-deficit-en-decarboxylase-dacide-l-amine-aromatique","status":"publish","type":"post","link":"https:\/\/rvh-synergie.org\/actualites\/la-fda-approuve-kebilidi-pour-le-deficit-en-decarboxylase-dacide-l-amine-aromatique\/","title":{"rendered":"La FDA approuve Kebilidi pour le d\u00e9ficit en d\u00e9carboxylase d&rsquo;acide L-amin\u00e9 aromatique"},"content":{"rendered":"<div>\n<div class=\"article-gallery lightGallery\">\n<div data-thumb=\"https:\/\/scx1.b-cdn.net\/csz\/news\/tmb\/2024\/fda-approves-kebilidi.jpg\" data-src=\"https:\/\/scx2.b-cdn.net\/gfx\/news\/2024\/fda-approves-kebilidi.jpg\" data-sub-html=\"\">\n<figure class=\"article-img\">\n<\/figure><\/div>\n<\/div>\n<p>La Food and Drug Administration des \u00c9tats-Unis a approuv\u00e9 Kebilidi (eladocagene exuparvovec-tneq) de PTC Therapeutics pour le traitement des patients adultes et p\u00e9diatriques pr\u00e9sentant un d\u00e9ficit en L-aminoacide d\u00e9carboxylase aromatique (AADC).<\/p>\n<p>La th\u00e9rapie g\u00e9nique bas\u00e9e sur un vecteur viral ad\u00e9no-associ\u00e9 est la premi\u00e8re th\u00e9rapie g\u00e9nique approuv\u00e9e par la FDA pour le traitement du d\u00e9ficit en AADC, une maladie g\u00e9n\u00e9tique rare affectant la production de certains neurotransmetteurs.<\/p>\n<p>Kebilidi doit \u00eatre administr\u00e9 dans un centre m\u00e9dical sp\u00e9cialis\u00e9 en neurochirurgie st\u00e9r\u00e9otaxique p\u00e9diatrique. Au cours d&rsquo;une s\u00e9ance chirurgicale, quatre perfusions sont administr\u00e9es dans la r\u00e9gion du cerveau impliqu\u00e9e dans le contr\u00f4le moteur.<\/p>\n<p>L&rsquo;approbation acc\u00e9l\u00e9r\u00e9e \u00e9tait bas\u00e9e sur une \u00e9tude clinique ouverte \u00e0 un seul groupe incluant 13 patients p\u00e9diatriques avec un diagnostic confirm\u00e9 de d\u00e9ficit en AADC.<\/p>\n<p>Au d\u00e9part, tous les patients n\u2019avaient aucune fonction motrice globale (la pr\u00e9sentation la plus grave du d\u00e9ficit en AADC) et une diminution de l\u2019activit\u00e9 de l\u2019AADC dans le plasma. Par rapport aux patients non trait\u00e9s (histoire naturelle), huit des 12 patients trait\u00e9s par Kebilidi ont pr\u00e9sent\u00e9 une am\u00e9lioration de la fonction motrice globale \u00e0 la semaine 48.<\/p>\n<p>\u00ab\u00a0Les progr\u00e8s cliniques dans le domaine de la th\u00e9rapie g\u00e9nique continuent de conduire \u00e0 la d\u00e9couverte et \u00e0 la disponibilit\u00e9 d&rsquo;options de traitement innovantes pour des maladies rares qui seraient autrement difficiles \u00e0 g\u00e9rer\u00a0\u00bb, a d\u00e9clar\u00e9 Peter Marks, MD, Ph.D., du Centre d&rsquo;\u00e9valuation et d&rsquo;\u00e9valuation des produits biologiques de la FDA. Recherche, a d\u00e9clar\u00e9 dans un communiqu\u00e9.<\/p>\n<p>                                        <!-- print only --><\/p><\/div>\n","protected":false},"excerpt":{"rendered":"<p>La Food and Drug Administration des \u00c9tats-Unis a approuv\u00e9 Kebilidi (eladocagene exuparvovec-tneq) de PTC Therapeutics<\/p>\n","protected":false},"author":1,"featured_media":2457,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[86],"tags":[321,2758,2756,2755,2754,447,2753,2757,8],"class_list":["post-2456","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-les-maladies","tag-approuve","tag-aromatique","tag-dacide","tag-decarboxylase","tag-deficit","tag-fda","tag-kebilidi","tag-lamine","tag-pour","generate-columns","tablet-grid-50","mobile-grid-100","grid-parent","grid-50","resize-featured-image"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v23.4 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>La FDA approuve Kebilidi pour le d\u00e9ficit en d\u00e9carboxylase d&#039;acide L-amin\u00e9 aromatique - RVH-Synergie.org<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/rvh-synergie.org\/actualites\/la-fda-approuve-kebilidi-pour-le-deficit-en-decarboxylase-dacide-l-amine-aromatique\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"La FDA approuve Kebilidi pour le d\u00e9ficit en d\u00e9carboxylase d&#039;acide L-amin\u00e9 aromatique - RVH-Synergie.org\" \/>\n<meta property=\"og:description\" content=\"La Food and Drug Administration des \u00c9tats-Unis a approuv\u00e9 Kebilidi (eladocagene exuparvovec-tneq) de PTC Therapeutics\" \/>\n<meta property=\"og:url\" content=\"https:\/\/rvh-synergie.org\/actualites\/la-fda-approuve-kebilidi-pour-le-deficit-en-decarboxylase-dacide-l-amine-aromatique\/\" \/>\n<meta property=\"og:site_name\" content=\"RVH-Synergie\" \/>\n<meta property=\"article:published_time\" content=\"2024-11-27T12:55:49+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2024-11-27T12:55:50+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/rvh-synergie.org\/actualites\/wp-content\/uploads\/2024\/11\/1732712149_La-FDA-approuve-Kebilidi-pour-le-deficit-en-decarboxylase-dacide.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"1280\" \/>\n\t<meta property=\"og:image:height\" content=\"853\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"Charles Martinez\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"\u00c9crit par\" \/>\n\t<meta name=\"twitter:data1\" content=\"Charles Martinez\" \/>\n\t<meta name=\"twitter:label2\" content=\"Dur\u00e9e de lecture estim\u00e9e\" \/>\n\t<meta name=\"twitter:data2\" content=\"1 minute\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/rvh-synergie.org\/actualites\/la-fda-approuve-kebilidi-pour-le-deficit-en-decarboxylase-dacide-l-amine-aromatique\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/rvh-synergie.org\/actualites\/la-fda-approuve-kebilidi-pour-le-deficit-en-decarboxylase-dacide-l-amine-aromatique\/\"},\"author\":{\"name\":\"Charles Martinez\",\"@id\":\"https:\/\/rvh-synergie.org\/actualites\/#\/schema\/person\/aa5ea98671fb4e8620f488a0795d2560\"},\"headline\":\"La FDA approuve Kebilidi pour le d\u00e9ficit en d\u00e9carboxylase d&rsquo;acide L-amin\u00e9 aromatique\",\"datePublished\":\"2024-11-27T12:55:49+00:00\",\"dateModified\":\"2024-11-27T12:55:50+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/rvh-synergie.org\/actualites\/la-fda-approuve-kebilidi-pour-le-deficit-en-decarboxylase-dacide-l-amine-aromatique\/\"},\"wordCount\":298,\"publisher\":{\"@id\":\"https:\/\/rvh-synergie.org\/actualites\/#organization\"},\"image\":{\"@id\":\"https:\/\/rvh-synergie.org\/actualites\/la-fda-approuve-kebilidi-pour-le-deficit-en-decarboxylase-dacide-l-amine-aromatique\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/rvh-synergie.org\/actualites\/wp-content\/uploads\/2024\/11\/1732712149_La-FDA-approuve-Kebilidi-pour-le-deficit-en-decarboxylase-dacide.jpg\",\"keywords\":[\"approuve\",\"aromatique\",\"dacide\",\"d\u00e9carboxylase\",\"d\u00e9ficit\",\"FDA\",\"Kebilidi\",\"Lamin\u00e9\",\"pour\"],\"articleSection\":[\"Les maladies\"],\"inLanguage\":\"fr-FR\"},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/rvh-synergie.org\/actualites\/la-fda-approuve-kebilidi-pour-le-deficit-en-decarboxylase-dacide-l-amine-aromatique\/\",\"url\":\"https:\/\/rvh-synergie.org\/actualites\/la-fda-approuve-kebilidi-pour-le-deficit-en-decarboxylase-dacide-l-amine-aromatique\/\",\"name\":\"La FDA approuve Kebilidi pour le d\u00e9ficit en d\u00e9carboxylase d'acide L-amin\u00e9 aromatique - 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