{"id":3477,"date":"2025-04-01T17:31:55","date_gmt":"2025-04-01T14:31:55","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3477"},"modified":"2025-04-04T10:01:49","modified_gmt":"2025-04-04T07:01:49","slug":"three-ongoing-clinical-trials-of-elevidys-gene-therapy-temporarily-halted-in-europe","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/es\/three-ongoing-clinical-trials-of-elevidys-gene-therapy-temporarily-halted-in-europe\/","title":{"rendered":"Tres ensayos cl\u00ednicos en curso de la terapia g\u00e9nica Elevidys se suspenden temporalmente en Europa"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Tres ensayos cl\u00ednicos en curso de ELEVIDYS (delandistr\u00f3geno moxeparvovec-rokl), una terapia g\u00e9nica desarrollada por Sarepta y comercializada por Roche, han sido suspendidos temporalmente por la Agencia Europea de Medicamentos (EMA). Esta noticia, esperada pero no inoportuna, ha causado profunda tristeza en la comunidad de pacientes con distrofia muscular de Duchenne (DMD).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Los ensayos pausados incluyen el estudio de fase 3 Envision patrocinado por Sarepta, que eval\u00faa Elevidys en ni\u00f1os de 8 a 17 a\u00f1os; el estudio de fase 2 Envol de Roche, que investiga Elevidys en beb\u00e9s y reci\u00e9n nacidos; y el Estudio 104 de Sarepta, una fase 1 que analiza Elevidys en ni\u00f1os de 4 a 9 a\u00f1os con anticuerpos preexistentes a un serotipo particular.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Antes de Roche, Sarepta y EMA, la primera comunidad en publicar este anuncio fue nuestro socio turco &gt;&gt;&gt; <a href=\"https:\/\/dmd.arti.net\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD Dayanisma<\/a>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Roche ha emitido una carta a la comunidad que pod\u00e9is leer aqu\u00ed. (<a href=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/March-2025-Roche-DMD-Temporary-Clinical-Study-Halt-Community-Letter_31Mar25.pdf\" target=\"_blank\" rel=\"noreferrer noopener\">PDF<\/a>)<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>Tabla de contenido<\/h2><nav><ul><li><a href=\"#why-were-elevidys-clinical-trials-halted\">\u00bfPor qu\u00e9 se detuvieron los ensayos cl\u00ednicos de Elevidys?<\/a><ul><li><a href=\"#srp-9001-104\">SRP-9001-104 Suspendido temporalmente<\/a><\/li><li><a href=\"#envol-study-302\">ENVOL (Estudio 302) Suspendido Temporalmente<\/a><\/li><li><a href=\"#srp-9001-303\">SRP-9001-303 Suspendido temporalmente<\/a><\/li><\/ul><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"why-were-elevidys-clinical-trials-halted\">\u00bfPor qu\u00e9 se detuvieron los ensayos cl\u00ednicos de Elevidys?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Tras la muerte de un joven de 16 a\u00f1os con distrofia muscular de Duchenne que recibi\u00f3 una infusi\u00f3n de Elevidys el 18 de marzo de 2025, las perspectivas para la terapia g\u00e9nica, que se comercializa a un precio comercial de $3 millones, cambiaron abruptamente.<a href=\"https:\/\/dmdwarrior.com\/es\/sixteen-year-old-dies-after-receiving-sareptas-duchenne-gene-therapy-elevidys\/\">Leer m\u00e1s<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Hasta que se determine la causa precisa de la muerte de un adolescente estadounidense que desarroll\u00f3 insuficiencia hep\u00e1tica aguda (IAA) tras tomar Elevidys, la Agencia Europea de Medicamentos (EMA) solicit\u00f3 la suspensi\u00f3n de los estudios. Tras el fallecimiento del paciente el mes pasado, Sarepta anunci\u00f3 que modificar\u00e1 la etiqueta del Elevidys para incluir la advertencia de seguridad.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"srp-9001-104\">SRP-9001-104 Suspendido temporalmente<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"762\" height=\"113\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED.jpg\" alt=\"El ensayo cl\u00ednico SRP-9001-104 de la terapia g\u00e9nica Elevidys se suspende temporalmente en Europa\" class=\"wp-image-3480\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED.jpg 762w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-300x44.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-18x3.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-150x22.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-696x103.jpg 696w\" sizes=\"auto, (max-width: 762px) 100vw, 762px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Fuente: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2024-512624-11-00?lang=en\" target=\"_blank\" rel=\"noopener\">Ensayo cl\u00ednico de SRP-9001-104<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"envol-study-302\">ENVOL (Estudio 302) Suspendido Temporalmente<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"762\" height=\"272\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted.jpg\" alt=\"El ensayo cl\u00ednico ENVOL (Estudio 302) de la terapia g\u00e9nica Elevidys se suspende temporalmente en Europa\" class=\"wp-image-3481\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted.jpg 762w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-300x107.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-18x6.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-150x54.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-696x248.jpg 696w\" sizes=\"auto, (max-width: 762px) 100vw, 762px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Fuente: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2023-509901-57-00?lang=en\" target=\"_blank\" rel=\"noopener\">Ensayo cl\u00ednico de SRP-9001-302<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"srp-9001-303\">SRP-9001-303 Suspendido temporalmente<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"768\" height=\"313\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted.jpg\" alt=\"El ensayo cl\u00ednico SRP-9001-303 de la terapia g\u00e9nica Elevidys se suspende temporalmente en Europa\" class=\"wp-image-3482\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-300x122.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-18x7.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-150x61.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-696x284.jpg 696w\" sizes=\"auto, (max-width: 768px) 100vw, 768px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Fuente: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2024-512626-28-00?lang=en\" target=\"_blank\" rel=\"noopener\">Ensayo cl\u00ednico de SRP-9001-303<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">M\u00e1s informaci\u00f3n: <a href=\"https:\/\/dmdwarrior.com\/es\/dmd-warriors-turkish-representative-shares-his-views-on-elevidys-gene-therapy-is-it-effective-and-why-is-it-expensive\/\">El representante turco de DMD WarrioR comparte sus puntos de vista sobre la terapia gen\u00e9tica Elevidys.<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Three ongoing clinical trials of ELEVIDYS (delandistrogene moxeparvovec-rokl), a gene therapy developed by Sarepta and marketed by Roche, have been temporarily halted by the European Medicines Agency (EMA). This expected but not unwelcome news has caused deep sadness in the Duchenne muscular dystrophy (DMD) community. The paused trials include the Sarepta-sponsored phase 3 Envision study, [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3478,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[242,33,179,91,84,213,61,203,65],"class_list":["post-3477","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-clinical-trials","tag-elevidys","tag-elevidys-gene-therapy","tag-ema","tag-europe","tag-european-medicines-agency","tag-gene-therapy","tag-roche","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/3477","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/comments?post=3477"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/3477\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media\/3478"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media?parent=3477"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/categories?post=3477"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/tags?post=3477"}],"curies":[{"name":"gracias","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}