{"id":6196,"date":"2026-02-09T17:21:36","date_gmt":"2026-02-09T14:21:36","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6196"},"modified":"2026-02-09T17:21:38","modified_gmt":"2026-02-09T14:21:38","slug":"solid-biosciences-aligns-with-fda-on-sgt-003-phase-3-trial-design-for-duchenne-muscular-dystrophy-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/pt\/solid-biosciences-aligns-with-fda-on-sgt-003-phase-3-trial-design-for-duchenne-muscular-dystrophy-gene-therapy\/","title":{"rendered":"A variante Solid Biosciences alinha-se com a FDA no desenho do ensaio cl\u00ednico de fase 3 SGT-003 para terapia g\u00eanica da distrofia muscular de Duchenne."},"content":{"rendered":"<p class=\"wp-block-paragraph\">A Solid Biosciences anunciou uma atualiza\u00e7\u00e3o regulat\u00f3ria positiva de sua recente reuni\u00e3o do Tipo C com a Food and Drug Administration (FDA) dos EUA (FDA), que apoia o avan\u00e7o cont\u00ednuo do SGT-003 como um tratamento potencial para a distrofia muscular de Duchenne.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>IMPACT DUCHENNE: Empresa alinhada com a FDA no desenho de um estudo de Fase 3 randomizado, duplo-cego e controlado por placebo \u2013<\/li>\n\n\n\n<li>IMPACT DUCHENNE: Empresa prev\u00ea administra\u00e7\u00e3o da primeira dose a participantes no primeiro trimestre de 2026 \u2013<\/li>\n\n\n\n<li>A empresa planeja realizar reuni\u00f5es adicionais com o FDA no primeiro semestre de 2026 para alinhar um poss\u00edvel caminho de aprova\u00e7\u00e3o acelerada para o SGT-003.<\/li>\n\n\n\n<li>INSPIRE DUCHENNE: O SGT-003 continua sendo geralmente bem tolerado, com 36 participantes que receberam a dose at\u00e9 9 de fevereiro de 2026, no ensaio cl\u00ednico de Fase 1\/2.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">O comit\u00ea FDA concordou que o desenho do estudo era razo\u00e1vel, incluindo: a popula\u00e7\u00e3o de pacientes ambulantes com idade entre 7 e &lt;12 anos, o desfecho prim\u00e1rio de mudan\u00e7a em rela\u00e7\u00e3o ao valor basal na velocidade de Tempo para Levantar (TTR) da posi\u00e7\u00e3o supina avaliada em 18 meses e outros desfechos secund\u00e1rios importantes.<\/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>Solid Biosciences announced a positive regulatory update from its recent Type C meeting with the U.S. Food and Drug Administration (FDA) that supports the continued advancement of SGT-003 as a potential treatment for Duchenne muscular dystrophy. The FDA agreed that the trial design was reasonable including: the patient population of ambulant participants 7 to &lt;12 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6197,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[88,110,170,109],"class_list":["post-6196","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-fda","tag-sgt-003","tag-sgt-003-gene-therapy","tag-solid-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/6196","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=6196"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/6196\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/6197"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=6196"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=6196"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=6196"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}