{"id":4108,"date":"2025-07-16T10:53:45","date_gmt":"2025-07-16T07:53:45","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4108"},"modified":"2025-07-16T10:53:47","modified_gmt":"2025-07-16T07:53:47","slug":"sarepta-therapeutics-halts-elevidys-shipments-for-non-ambulatory-dmd-patients","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/pt\/sarepta-therapeutics-halts-elevidys-shipments-for-non-ambulatory-dmd-patients\/","title":{"rendered":"Sarepta Therapeutics suspende remessas de ELEVIDYS para pacientes com DMD n\u00e3o ambulatoriais"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Devido a dois incidentes documentados de insufici\u00eancia hep\u00e1tica grave que resultaram em morte, a Sarepta Therapeutics interrompeu o envio de seu tratamento gen\u00e9tico ELEVIDYS para indiv\u00edduos com distrofia muscular de Duchenne n\u00e3o ambulat\u00f3ria.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">O paciente que faleceu mais recentemente tinha 15 anos. Para permitir mudan\u00e7as no protocolo e considera\u00e7\u00f5es regulat\u00f3rias, a Sarepta decidiu interromper o estudo confirmat\u00f3rio de Fase 3 do ENVISION (SRP-9001-303), que envolve adultos ambulatoriais e n\u00e3o ambulatoriais, devido a esses eventos adversos graves. <strong>Ler mais<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/pt\/three-ongoing-clinical-trials-of-elevidys-gene-therapy-temporarily-halted-in-europe\/\">Tr\u00eas ensaios cl\u00ednicos em andamento da terapia gen\u00e9tica Elevidys foram temporariamente interrompidos na Europa<\/a> \u2013<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Os dois casos fatais foram relatados em junho de 2025, com o FDA emitindo um comunicado de seguran\u00e7a em 24 de junho de 2025, para investigar o risco potencial de insufici\u00eancia hep\u00e1tica aguda associada ao ELEVIDYS. Em resposta, a Sarepta interrompeu seu estudo de Fase 3, ENVISION, para atualizar os protocolos e consultar os \u00f3rg\u00e3os reguladores. A empresa tamb\u00e9m suspendeu temporariamente a distribui\u00e7\u00e3o do ELEVIDYS para pacientes n\u00e3o deambulantes at\u00e9 que um novo regime imunossupressor seja aprovado.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Saber mais<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/pt\/aan-report-reviews-the-state-of-evidence-on-elevidys-gene-therapy-cure-for-duchenne-muscular-dystrophy\/\">Ap\u00f3s a morte do segundo paciente, comunidades com distrofia muscular de Duchenne querem respostas sobre a terapia gen\u00e9tica Elevidys<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Due to two documented incidents of severe liver failure that resulted in death, Sarepta Therapeutics has stopped shipping its gene treatment ELEVIDYS for individuals with non-ambulatory Duchenne muscular dystrophy. The patient who died most recently was 15 years old. In order to allow for protocol changes and regulatory considerations, Sarepta has decided to halt the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4109,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[33,65],"class_list":["post-4108","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-elevidys","tag-sarepta"],"subtitle":"A Sarepta suspendeu temporariamente as remessas de ELEVIDYS para pacientes com distrofia muscular de Duchenne (DMD) n\u00e3o deambuladores ap\u00f3s as mortes relatadas de dois pacientes que apresentaram insufici\u00eancia hep\u00e1tica aguda ap\u00f3s tratamento com o medicamento.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4108","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=4108"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4108\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/4109"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=4108"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=4108"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=4108"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}