{"id":7875,"date":"2026-05-08T10:37:41","date_gmt":"2026-05-08T07:37:41","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=7875"},"modified":"2026-05-08T10:37:43","modified_gmt":"2026-05-08T07:37:43","slug":"bmn-351-exon-51-skipping-therapy-phase-2-clinical-trial-nct07573631","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/pt\/bmn-351-exon-51-skipping-therapy-phase-2-clinical-trial-nct07573631\/","title":{"rendered":"A BioMarin inicia o ensaio cl\u00ednico de fase 2 da terapia de supress\u00e3o do \u00e9xon 51 com BMN 351 para distrofia muscular de Duchenne."},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>A BioMarin Pharmaceutical lan\u00e7ou oficialmente um novo estudo de extens\u00e3o de Fase 2 (NCT07573631) para avaliar o BMN 351, uma terapia experimental de supress\u00e3o do \u00e9xon 51, desenvolvida para pessoas com distrofia muscular de Duchenne (DMD).<\/strong> O estudo se concentrar\u00e1 na seguran\u00e7a a longo prazo, tolerabilidade e resultados da fun\u00e7\u00e3o f\u00edsica em participantes que j\u00e1 conclu\u00edram o ensaio cl\u00ednico anterior 351-201.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">O que \u00e9 o ensaio cl\u00ednico de fase 2 da terapia de supress\u00e3o do \u00e9xon 51 com BMN 351?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>O ensaio cl\u00ednico de fase 2 da terapia de exclus\u00e3o do \u00e9xon 51 com BMN 351 \u00e9 um estudo de extens\u00e3o multic\u00eantrico e aberto para indiv\u00edduos com distrofia muscular de Duchenne que s\u00e3o eleg\u00edveis para a exclus\u00e3o do \u00e9xon 51.<\/strong> Os pesquisadores ir\u00e3o avaliar a seguran\u00e7a e a efic\u00e1cia das infus\u00f5es intravenosas semanais de BMN 351 ao longo de um per\u00edodo de tratamento mais prolongado.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">O ensaio cl\u00ednico planeja recrutar at\u00e9 18 participantes com idades entre 4 e 23 anos que j\u00e1 conclu\u00edram o estudo de Fase 1\/2 conhecido como 351-201.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Entendendo a Distrofia Muscular de Duchenne e a S\u00edndrome de Omiss\u00e3o do Exon 51<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">O que \u00e9 a distrofia muscular de Duchenne?<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">A distrofia muscular de Duchenne \u00e9 uma doen\u00e7a gen\u00e9tica rara que causa fraqueza muscular progressiva e degenera\u00e7\u00e3o muscular. \u00c9 causada por muta\u00e7\u00f5es no gene da distrofina, essencial para a manuten\u00e7\u00e3o de c\u00e9lulas musculares saud\u00e1veis.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">O que est\u00e1 sendo pulado no \u00e9xon 51?<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">As terapias de exclus\u00e3o de \u00e9xons s\u00e3o projetadas para ajudar o corpo a produzir uma vers\u00e3o mais curta, por\u00e9m funcional, da prote\u00edna distrofina. A exclus\u00e3o do \u00e9xon 51 \u00e9 direcionada especificamente a pacientes cujas muta\u00e7\u00f5es podem se beneficiar da exclus\u00e3o do \u00e9xon 51 durante o processo de produ\u00e7\u00e3o da prote\u00edna.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">O BMN 351 est\u00e1 sendo investigado como uma terapia de pr\u00f3xima gera\u00e7\u00e3o para o salto de \u00e9xons, que pode ajudar a retardar a progress\u00e3o da doen\u00e7a em pacientes eleg\u00edveis.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"847\" height=\"1024\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-847x1024.jpg\" alt=\"Muta\u00e7\u00f5es e dele\u00e7\u00f5es pass\u00edveis de terapias de salto do exon 51 para distrofia muscular de Duchenne\" class=\"wp-image-4526\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-847x1024.jpg 847w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-248x300.jpg 248w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-768x929.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1270x1536.jpg 1270w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1693x2048.jpg 1693w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-10x12.jpg 10w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-347x420.jpg 347w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-150x181.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-300x363.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-696x842.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1068x1292.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1920x2323.jpg 1920w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping.jpg 1984w\" sizes=\"auto, (max-width: 847px) 100vw, 847px\" \/><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\">Detalhes importantes sobre o estudo de Fase 2<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">Desenho do estudo<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">O julgamento \u00e9:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Multic\u00eantrico<\/li>\n\n\n\n<li>R\u00f3tulo aberto<\/li>\n\n\n\n<li>estudo de extens\u00e3o de longo prazo<\/li>\n\n\n\n<li>Focado na administra\u00e7\u00e3o intravenosa semanal<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">A primeira visita deste estudo tamb\u00e9m servir\u00e1 como a visita final do ensaio cl\u00ednico anterior 351-201.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Cronograma estimado<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>In\u00edcio do estudo: abril de 2026<\/li>\n\n\n\n<li>Conclus\u00e3o prim\u00e1ria: dezembro de 2031<\/li>\n\n\n\n<li>Previs\u00e3o de conclus\u00e3o do estudo: dezembro de 2031<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">Pa\u00edses participantes do estudo<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">O estudo inclui locais de ensaios cl\u00ednicos em:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>It\u00e1lia<\/li>\n\n\n\n<li>Holanda<\/li>\n\n\n\n<li>Espanha<\/li>\n\n\n\n<li>Turquia<\/li>\n\n\n\n<li>Reino Unido<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">Quem pode participar?<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">Crit\u00e9rios principais de inclus\u00e3o<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Os participantes devem:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Conclu\u00ed o estudo 351-201.<\/li>\n\n\n\n<li>Continue o tratamento est\u00e1vel com corticosteroides.<\/li>\n\n\n\n<li>Siga os requisitos da visita de estudo<\/li>\n\n\n\n<li>Fornecer consentimento informado ou assentimento quando aplic\u00e1vel.<\/li>\n\n\n\n<li>Utilize contracep\u00e7\u00e3o, se exigido pelas diretrizes do protocolo.<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">Principais crit\u00e9rios de exclus\u00e3o<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Os participantes n\u00e3o podem participar se:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Apresentam dist\u00farbios de coagula\u00e7\u00e3o conhecidos.<\/li>\n\n\n\n<li>Usar medicamentos proibidos<\/li>\n\n\n\n<li>Recebeu outra terapia de exclus\u00e3o de \u00e9xon aprovada dentro de 12 semanas antes do in\u00edcio do estudo.<\/li>\n\n\n\n<li>J\u00e1 recebeu terapia g\u00eanica para DMD (Distrofia Muscular de Duchenne) anteriormente.<\/li>\n\n\n\n<li>Utilize anticoagulantes ou imunossupressores.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">Por que este julgamento \u00e9 importante para a comunidade de Duchenne?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>O lan\u00e7amento do ensaio cl\u00ednico de fase 2 da terapia de supress\u00e3o do \u00e9xon 51 BMN 351 representa mais um passo importante no desenvolvimento de terapias gen\u00e9ticas direcionadas para a distrofia muscular de Duchenne.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Estudos de extens\u00e3o de longo prazo s\u00e3o especialmente importantes porque ajudam os pesquisadores a compreender:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Se os benef\u00edcios do tratamento podem ser mantidos ao longo do tempo.<\/li>\n\n\n\n<li>Perfis de seguran\u00e7a a longo prazo<\/li>\n\n\n\n<li>Efeitos sobre a fun\u00e7\u00e3o f\u00edsica di\u00e1ria<\/li>\n\n\n\n<li>Potencial impacto na progress\u00e3o da doen\u00e7a<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Para as fam\u00edlias afetadas pela distrofia muscular de Duchenne, o progresso cont\u00ednuo nas pesquisas sobre o salto de \u00e9xons oferece uma esperan\u00e7a renovada para futuras op\u00e7\u00f5es de tratamento.<\/p>\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>BioMarin Pharmaceutical has officially launched a new Phase 2 extension study (NCT07573631) evaluating BMN 351, an investigational exon 51 skipping therapy designed for people living with Duchenne muscular dystrophy (DMD). The study will focus on long-term safety, tolerability, and physical function outcomes in participants who previously completed the earlier 351-201 clinical trial. What Is the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":7877,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[759,760,137,122,193,421,194],"class_list":["post-7875","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-biomarin","tag-bmn-351","tag-clinical-trial","tag-exon-51","tag-exon-51-skipping","tag-exon-51-skipping-therapy","tag-exon-51-treatment"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/7875","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=7875"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/7875\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/7877"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=7875"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=7875"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=7875"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}