{"id":4274,"date":"2025-08-04T17:03:02","date_gmt":"2025-08-04T14:03:02","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4274"},"modified":"2025-08-04T17:13:24","modified_gmt":"2025-08-04T14:13:24","slug":"dyne-therapeutics-announces-fda-breakthrough-therapy-designation-for-dyne-251-in-duchenne-muscular-dystrophy-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/pt\/dyne-therapeutics-announces-fda-breakthrough-therapy-designation-for-dyne-251-in-duchenne-muscular-dystrophy-dmd\/","title":{"rendered":"Dyne Therapeutics anuncia a designa\u00e7\u00e3o de terapia inovadora FDA para DYNE-251 na distrofia muscular de Duchenne (DMD)"},"content":{"rendered":"<p class=\"wp-block-paragraph\">A Dyne Therapeutics anunciou que a Food and Drug Administration (FDA) dos EUA concedeu a Designa\u00e7\u00e3o de Terapia Inovadora ao DYNE-251 para o tratamento de pacientes com distrofia muscular de Duchenne (DMD), pass\u00edveis de salto do exon 51. A designa\u00e7\u00e3o se baseia em dados do ensaio cl\u00ednico DELIVER em andamento.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">DYNE-251: Designa\u00e7\u00e3o de Terapia Inovadora<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cEsta Designa\u00e7\u00e3o de Terapia Inovadora para DYNE-251 \u00e9 uma prova de seu potencial como terapia de pr\u00f3xima gera\u00e7\u00e3o, projetada para trazer melhora funcional significativa a indiv\u00edduos com DMD, para os quais o salto do exon 51 pode levar \u00e0 produ\u00e7\u00e3o de distrofina quase completa\u201d, disse Doug Kerr, MD, Ph.D., diretor m\u00e9dico da Dyne. \u201cComo divulgamos anteriormente, DYNE-251 demonstrou melhora funcional sustentada ao longo de dezoito meses, avaliada por medidas-chave como tempo para se levantar e percentil 95 da velocidade da passada. O n\u00edvel de express\u00e3o de distrofina quase completa observado marca um avan\u00e7o significativo para potencialmente proporcionar benef\u00edcios significativos aos pacientes.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Saber mais<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/pt\/exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Pr\u00f3ximas terapias de salto do Exon 51 para o tratamento da distrofia muscular de Duchenne<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ler mais<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/pt\/dyne-therapeutics-receives-european-medicines-agency-ema-orphan-drug-designation-for-dyne-251-in-duchenne-muscular-dystrophy\/\">Dyne Therapeutics recebe designa\u00e7\u00e3o de medicamento \u00f3rf\u00e3o da Ag\u00eancia Europeia de Medicamentos (EMA) para DYNE-251 na distrofia muscular de Duchenne<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-251 for the treatment of patients with Duchenne muscular dystrophy (DMD), amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER clinical trial. DYNE-251: Breakthrough Therapy Designation \u201cThis Breakthrough Therapy Designation for DYNE-251 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4275,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[208,145,122,193,194,88],"class_list":["post-4274","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-fda"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4274","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=4274"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4274\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/4275"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=4274"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=4274"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=4274"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}