{"id":7601,"date":"2025-06-29T06:56:04","date_gmt":"2025-06-29T04:56:04","guid":{"rendered":"https:\/\/rvh-synergie.org\/actualites\/therapie-genique-efficace-chez-les-patients-atteints-depidermolyse-dystrophique-recessive-bullosa\/"},"modified":"2025-06-29T06:56:05","modified_gmt":"2025-06-29T04:56:05","slug":"therapie-genique-efficace-chez-les-patients-atteints-depidermolyse-dystrophique-recessive-bullosa","status":"publish","type":"post","link":"https:\/\/rvh-synergie.org\/actualites\/therapie-genique-efficace-chez-les-patients-atteints-depidermolyse-dystrophique-recessive-bullosa\/","title":{"rendered":"Th\u00e9rapie g\u00e9nique efficace chez les patients atteints d&rsquo;\u00e9pidermolyse dystrophique r\u00e9cessive Bullosa"},"content":{"rendered":"<div>\n<div class=\"article-gallery lightGallery\">\n<div data-thumb=\"https:\/\/scx1.b-cdn.net\/csz\/news\/tmb\/2025\/gene-therapy-effective.jpg\" data-src=\"https:\/\/scx2.b-cdn.net\/gfx\/news\/2025\/gene-therapy-effective.jpg\" data-sub-html=\"\">\n<figure class=\"article-img\">\n<\/figure><\/div>\n<\/div>\n<p>For patients with recessive dystrophic epidermolysis bullosa (RDEB), a one-time surgical application of prademagene zamikeracel, an autologous COL7A1 gene-modified cellular sheet that is sutured onto a large, chronic RDEB wound, improves wound healing and pain versus standard of care, according to a study published online June 23 in <i>Le lancet<\/i>.<\/p>\n<p>Jean Y. Tang, MD, Ph.D., de la Stanford University School of Medicine en Californie, et des coll\u00e8gues ont men\u00e9 un essai randomis\u00e9 en ouvert, de phase 3 men\u00e9 dans deux institutions pour examiner l&rsquo;efficacit\u00e9 et la s\u00e9curit\u00e9 d&rsquo;une application chirurgicale unique de Prademagene Zamikeracel dans la cicatrisation des plaies. Les patients \u00e9ligibles \u00e9taient \u00e2g\u00e9s de 6 ans ou plus, avec un diagnostic clinique et g\u00e9n\u00e9tique confirm\u00e9 du RDEB, et au moins deux plaies chroniques. De grandes plaies chroniques sur les participants ont \u00e9t\u00e9 appari\u00e9es par paires et assign\u00e9es au hasard au traitement avec Prademagene Zamikeracel ou contr\u00f4le (standard de soins).<\/p>\n<p>Onze patients ont \u00e9t\u00e9 inscrits, avec 43 paires de plaies randomis\u00e9es. Les chercheurs ont constat\u00e9 qu&rsquo;\u00e0 la semaine 24, 81% et 16% des blessures trait\u00e9es et t\u00e9moins, respectivement, ont \u00e9t\u00e9 gu\u00e9ries au moins 50% par rapport \u00e0 la ligne de base (diff\u00e9rence moyenne, 67%). De la ligne de base \u00e0 la semaine 24, le changement moyen de la douleur des plaies \u00e9tait de \u22123,07 et \u22120,90 chez ceux qui recevaient respectivement le Prademagene Zamikeracel et les t\u00e9moins (diff\u00e9rence moyenne par paire, \u22122,23). Aucun \u00e9v\u00e9nement ind\u00e9sirable grave li\u00e9 au traitement n&rsquo;a \u00e9t\u00e9 signal\u00e9.<\/p>\n<p>\u00ab\u00a0Avec notre nouvelle technique de th\u00e9rapie g\u00e9nique, nous avons r\u00e9ussi \u00e0 traiter les blessures les plus difficiles \u00e0 g\u00e9rer, qui \u00e9taient g\u00e9n\u00e9ralement aussi les plus douloureuses pour ces patients\u00a0\u00bb, a d\u00e9clar\u00e9 Tang dans un communiqu\u00e9.<\/p>\n<p>Plusieurs auteurs ont r\u00e9v\u00e9l\u00e9 des liens avec des soci\u00e9t\u00e9s biopharmaceutiques, notamment Abeona Therapeutics, qui a financ\u00e9 l&rsquo;\u00e9tude. Le brevet de Prademagene Zamikeracel est autoris\u00e9 \u00e0 Abeona.<\/p>\n<p>                                        <!-- print only --><\/p><\/div>\n","protected":false},"excerpt":{"rendered":"<p>For patients with recessive dystrophic epidermolysis bullosa (RDEB), a one-time surgical application of prademagene zamikeracel,<\/p>\n","protected":false},"author":1,"featured_media":7602,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[86],"tags":[282,5841,56,5838,5839,206,1064,49,616,5840,1063],"class_list":["post-7601","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-les-maladies","tag-atteints","tag-bullosa","tag-chez","tag-depidermolyse","tag-dystrophique","tag-efficace","tag-genique","tag-les","tag-patients","tag-recessive","tag-therapie","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 - 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