{"id":4855,"date":"2025-11-01T22:09:30","date_gmt":"2025-11-01T19:09:30","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4855"},"modified":"2025-11-02T11:17:33","modified_gmt":"2025-11-02T08:17:33","slug":"becker-muscular-dystrophy-eligible-for-elevidys-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/pt\/becker-muscular-dystrophy-eligible-for-elevidys-gene-therapy\/","title":{"rendered":"Pacientes com doen\u00e7a de Becker podem receber a terapia Elevidys? A terapia Elevidys beneficia pacientes com doen\u00e7a de Becker?"},"content":{"rendered":"<p class=\"wp-block-paragraph\">A distrofia muscular de Becker \u00e9 eleg\u00edvel para o Elevidys? Neste artigo, exploraremos as pesquisas atuais, as diretrizes para o FDA e as opini\u00f5es de especialistas para responder se a distrofia muscular de Becker \u00e9 eleg\u00edvel para o Elevidys e o que isso significa para os futuros avan\u00e7os da terapia g\u00eanica.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>A muta\u00e7\u00e3o Elevidys beneficia pacientes com doen\u00e7a de Becker?<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">N\u00e3o \u2014 Com base em informa\u00e7\u00f5es da literatura publicada, pacientes com distrofia muscular de Becker (DMB) podem n\u00e3o se beneficiar da terapia g\u00eanica com ELEVIDYS (delandistrogene moxeparvovec-rokl).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Segue uma explica\u00e7\u00e3o detalhada dos motivos:<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u00cdndice<\/h2><nav><ul><li><a href=\"#\u2705-what-elevidys-is-approved-for\">\u2705 Para que serve o ELEVIDYS?<\/a><\/li><li><a href=\"#\ud83d\udeab-why-bmd-patients-not-eligible\">\ud83d\udeab Por que pacientes com DMO n\u00e3o s\u00e3o eleg\u00edveis<\/a><\/li><li><a href=\"#\ud83d\udccb-summary\">\ud83d\udccb Resumo<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"\u2705-what-elevidys-is-approved-for\"><strong>\u2705 Para que serve o ELEVIDYS?<\/strong><\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>ELEVIDYS \u00e9 aprovado pela Food and Drug Administration (FDA) dos EUA (FDA) apenas para pacientes com distrofia muscular de Duchenne (DMD) com pelo menos 4 anos de idade, que sejam capazes de andar e que tenham uma muta\u00e7\u00e3o confirmada no gene DMD. <a href=\"https:\/\/dmdwarrior.com\/pt\/fda-elevidys-gene-therapy-will-continue-to-be-used-in-ambulatory-dmd-patients\/\" target=\"_blank\" rel=\"noreferrer noopener\">A terapia g\u00eanica com Elevidys continuar\u00e1 sendo usada em pacientes ambulatoriais com DMD.<\/a><\/li>\n\n\n\n<li>A comprova\u00e7\u00e3o do benef\u00edcio cl\u00ednico pode ser necess\u00e1ria para que o ELEVIDYS continue aprovado para pacientes n\u00e3o ambulantes com distrofia muscular de Duchenne. Estudos est\u00e3o em andamento para avaliar esse impacto potencial. <a href=\"https:\/\/www.elevidys.com\/treatment-journey\/eligibility\" target=\"_blank\" rel=\"noreferrer noopener\">Elevidys<\/a><\/li>\n\n\n\n<li>A bula do medicamento especifica que pacientes com qualquer dele\u00e7\u00e3o no \u00e9xon 8 e\/ou no \u00e9xon 9 do gene DMD t\u00eam contraindica\u00e7\u00e3o para o uso de ELEVIDYS. <a href=\"https:\/\/www.elevidyshcp.com\/about-elevidys\/importance-of-treatment\" target=\"_blank\" rel=\"noreferrer noopener\">Elevidys<\/a><\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"\ud83d\udeab-why-bmd-patients-not-eligible\"><strong>\ud83d\udeab Por que pacientes com DMO n\u00e3o s\u00e3o eleg\u00edveis<\/strong><\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>A distrofia muscular de Becker (BMD) \u00e9 causada por muta\u00e7\u00f5es no mesmo gene da distrofia muscular de Duchenne (DMD), mas essas muta\u00e7\u00f5es normalmente permitem a produ\u00e7\u00e3o de alguma distrofina parcialmente funcional, levando a uma forma mais branda da doen\u00e7a em compara\u00e7\u00e3o com a DMD. <strong>Ler mais<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/pt\/becker-muscular-dystrophy-bmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Distrofia Muscular de Becker: Sintomas, Causas e Curas<\/a><\/li>\n\n\n\n<li>Segundo um comunicado, o documento de pol\u00edtica cl\u00ednica afirma claramente: \u201c\u00c9 improv\u00e1vel que pacientes com distrofia muscular de Becker (DMB) se beneficiem da terapia g\u00eanica ELEVIDYS.\u201d<a href=\"https:\/\/www.molinaclinicalpolicy.com\/molinaclinicalpolicy\/-\/media\/Molina\/PublicWebsite\/PDF\/Common\/Molina-Clinical-Policy\/Elevidys_delandistrogene-moxeparvovec_R.pdf\" target=\"_blank\" rel=\"noreferrer noopener\">Molina Healthcare<\/a>\u2026 <a href=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Elevidys_delandistrogene-moxeparvovec_R.pdf\" target=\"_blank\" rel=\"noreferrer noopener\">Leia isto<\/a>)<\/li>\n\n\n\n<li>A terapia foi desenvolvida para a defici\u00eancia mais grave na DMD, e n\u00e3o para a situa\u00e7\u00e3o de funcionamento parcial na BMD.<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"\ud83d\udccb-summary\">\ud83d\udccb Resumo<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Eleg\u00edvel<\/strong>: DMD, idade \u2265 4 anos e deambulante, muta\u00e7\u00e3o confirmada do gene DMD, excluindo certas dele\u00e7\u00f5es (exon 8\/9).<\/li>\n\n\n\n<li><strong>N\u00e3o eleg\u00edvel<\/strong>Pacientes com distrofia muscular de Becker (devido a diferentes mecanismos\/gravidade da doen\u00e7a) e aqueles com certas dele\u00e7\u00f5es do gene DMD (exons 8\/9).<\/li>\n\n\n\n<li>Se voc\u00ea tem Becker, o ELEVIDYS n\u00e3o est\u00e1 indicado na cobertura\/aprova\u00e7\u00e3o atual.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Saber mais<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/pt\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Perguntas frequentes sobre Elevidys<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Is Becker muscular dystrophy eligible for Elevidys? In this article, we\u2019ll explore current research, FDA guidance, and expert insights to answer whether Becker muscular dystrophy is eligible for Elevidys and what this means for future gene therapy advancements. Does Elevidys Benefit Becker Patients? No \u2014 Based on published literature information, patients with Becker muscular dystrophy [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4988,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[121,33,179,61,203,65],"class_list":["post-4855","post","type-post","status-publish","format-standard","has-post-thumbnail","category-dmd","tag-delandistrogene-moxeparvovec","tag-elevidys","tag-elevidys-gene-therapy","tag-gene-therapy","tag-roche","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4855","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=4855"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4855\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/4988"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=4855"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=4855"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=4855"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}