{"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\/pt\/dyne-therapeutics-to-apply-for-accelerated-approval-in-q2-2026-for-exon-51-skipping-therapy-dyne-251-z-rostudirsen\/","title":{"rendered":"Dyne Therapeutics solicitar\u00e1 aprova\u00e7\u00e3o acelerada no segundo trimestre de 2026 para a terapia de supress\u00e3o do \u00e9xon 51 (DYNE-251)."},"content":{"rendered":"<p class=\"wp-block-paragraph\">A Dyne Therapeutics anunciou resultados preliminares positivos da Coorte de Expans\u00e3o de Registro (REC) de seu estudo de Fase 1\/2 DELIVER, que avalia o zeleciment rostudirsen (z-rostudirsen, tamb\u00e9m conhecido como DYNE-251) em indiv\u00edduos com distrofia muscular de Duchenne (DMD) pass\u00edveis de exclus\u00e3o do \u00e9xon 51.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Descubra agora:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/pt\/mutations-and-deletions-amenable-to-exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Muta\u00e7\u00f5es e dele\u00e7\u00f5es pass\u00edveis de terapias de salto do exon 51<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Resultados do ensaio cl\u00ednico DYNE-251<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>A Coorte de Expans\u00e3o de Registro (REC) atingiu o objetivo prim\u00e1rio, demonstrando um aumento estatisticamente significativo na distrofina para 5,46% em 6 meses (ajustado para o conte\u00fado muscular; p&lt;0,0001), replicando a mesma altera\u00e7\u00e3o de 7 vezes em rela\u00e7\u00e3o ao valor basal observada anteriormente na dose de registro.<\/li>\n\n\n\n<li><strong>Observou-se melhora funcional em m\u00faltiplos par\u00e2metros cl\u00ednicos aos 6 meses no grupo REC; a fun\u00e7\u00e3o pulmonar foi preservada aos 6 meses.<\/strong><\/li>\n\n\n\n<li>Novos resultados positivos a longo prazo do estudo DELIVER demonstraram melhora funcional sustentada em todos os desfechos avaliados ao longo de 24 meses.<\/li>\n\n\n\n<li><strong>Perfil de seguran\u00e7a e tolerabilidade favor\u00e1vel cont\u00ednuo \u2013<\/strong><\/li>\n\n\n\n<li>O pedido de aprova\u00e7\u00e3o acelerada nos EUA est\u00e1 dentro do cronograma para o segundo trimestre de 2026 \u2013<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Siga esta p\u00e1gina<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/pt\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Todos os ensaios cl\u00ednicos para distrofia muscular 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\/pt\/wp-json\/wp\/v2\/posts\/5295","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/comments?post=5295"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/5295\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/5297"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=5295"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=5295"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=5295"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}