{"id":5295,"date":"2025-12-08T16:05:39","date_gmt":"2025-12-08T13:05:39","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5295"},"modified":"2025-12-08T16:08:05","modified_gmt":"2025-12-08T13:08:05","slug":"dyne-therapeutics-to-apply-for-accelerated-approval-in-q2-2026-for-exon-51-skipping-therapy-dyne-251-z-rostudirsen","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/dyne-therapeutics-to-apply-for-accelerated-approval-in-q2-2026-for-exon-51-skipping-therapy-dyne-251-z-rostudirsen\/","title":{"rendered":"Dyne Therapeutics demandera une approbation acc\u00e9l\u00e9r\u00e9e au deuxi\u00e8me trimestre 2026 pour la th\u00e9rapie par saut d&#039;exon 51 (DYNE-251)"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Dyne Therapeutics a annonc\u00e9 des r\u00e9sultats pr\u00e9liminaires positifs de la cohorte d&#039;expansion d&#039;enregistrement (REC) de son essai de phase 1\/2 DELIVER \u00e9valuant le zeleciment rostudirsen (z-rostudirsen, \u00e9galement connu sous le nom de DYNE-251) chez les personnes atteintes de dystrophie musculaire de Duchenne (DMD) pouvant b\u00e9n\u00e9ficier d&#039;un saut de l&#039;exon 51.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>D\u00e9couvrez maintenant :<\/strong> <a href=\"https:\/\/dmdwarrior.com\/fr\/mutations-and-deletions-amenable-to-exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Mutations et d\u00e9l\u00e9tions sensibles aux th\u00e9rapies par saut d&#039;exon 51<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">R\u00e9sultats de l&#039;essai clinique DYNE-251<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>La cohorte d&#039;expansion d&#039;enregistrement (REC) a atteint son crit\u00e8re d&#039;\u00e9valuation principal, d\u00e9montrant une augmentation statistiquement significative de la dystrophine \u00e0 5,46% \u00e0 6 mois (ajust\u00e9e \u00e0 la teneur musculaire ; p &lt; 0,0001), reproduisant la m\u00eame variation de 7 fois par rapport \u00e0 la valeur de r\u00e9f\u00e9rence pr\u00e9c\u00e9demment observ\u00e9e \u00e0 la dose d&#039;enregistrement \u2013<\/li>\n\n\n\n<li><strong>Une am\u00e9lioration fonctionnelle a \u00e9t\u00e9 observ\u00e9e pour plusieurs crit\u00e8res d&#039;\u00e9valuation cliniques \u00e0 6 mois dans le groupe REC ; la fonction pulmonaire a \u00e9t\u00e9 pr\u00e9serv\u00e9e \u00e0 6 mois.<\/strong><\/li>\n\n\n\n<li>De nouveaux r\u00e9sultats positifs \u00e0 long terme de l&#039;essai DELIVER ont montr\u00e9 une am\u00e9lioration fonctionnelle durable pour tous les crit\u00e8res d&#039;\u00e9valuation sur une p\u00e9riode de 24 mois.<\/li>\n\n\n\n<li><strong>Maintien d&#039;un profil de s\u00e9curit\u00e9 et de tol\u00e9rance favorable \u2013<\/strong><\/li>\n\n\n\n<li>Le d\u00e9p\u00f4t de la demande d&#039;approbation acc\u00e9l\u00e9r\u00e9e aux \u00c9tats-Unis est en bonne voie pour le deuxi\u00e8me trimestre 2026 \u2013<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Suivre cette page<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/fr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Tous les essais cliniques pour la dystrophie musculaire de Duchenne<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Dyne Therapeutics announced positive topline results from the Registrational Expansion Cohort (REC) of its Phase 1\/2 DELIVER trial evaluating zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Discover Now: Mutations and Deletions Amenable to Exon 51 Skipping Therapies Results of DYNE-251 Clinical Trial Follow [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5297,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[143,208,145,122,193,421,194,460],"class_list":["post-5295","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-skipping-therapy","tag-exon-51-treatment","tag-z-rostudirsen"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/5295","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/comments?post=5295"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/5295\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/5297"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=5295"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=5295"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=5295"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}