{"id":5183,"date":"2025-11-19T18:44:39","date_gmt":"2025-11-19T15:44:39","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5183"},"modified":"2025-11-19T19:06:37","modified_gmt":"2025-11-19T16:06:37","slug":"avidity-biosciences-announces-u-s-managed-access-program-map-for-investigational-therapy-del-zota-in-dmd44","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/avidity-biosciences-announces-u-s-managed-access-program-map-for-investigational-therapy-del-zota-in-dmd44\/","title":{"rendered":"Avidity Biosciences annonce un programme d&#039;acc\u00e8s g\u00e9r\u00e9 (MAP) aux \u00c9tats-Unis pour la th\u00e9rapie exp\u00e9rimentale del-zota (saut de l&#039;exon 44)"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Avidity Biosciences a annonc\u00e9 son programme d&#039;acc\u00e8s g\u00e9r\u00e9 (MAP) pour le traitement exp\u00e9rimental delpacibart zotadirsen (del-zota) pour les personnes \u00e9ligibles atteintes de mutations de la dystrophie musculaire de Duchenne pouvant \u00eatre modifi\u00e9es par saut de l&#039;exon 44 (DMD44) aux \u00c9tats-Unis.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201c Nous sommes conscients des besoins consid\u00e9rables de la communaut\u00e9 des personnes atteintes de DMD44, \u00e9tant donn\u00e9 l&#039;absence de th\u00e9rapies d&#039;exclusion d&#039;exons approuv\u00e9es pour cette maladie, ainsi que du potentiel du del-zota, mis en \u00e9vidence par les donn\u00e9es sans pr\u00e9c\u00e9dent de nos \u00e9tudes cliniques \u201d, a d\u00e9clar\u00e9 Sarah Boyce, pr\u00e9sidente-directrice g\u00e9n\u00e9rale d&#039;Avidity. \u201c Nous sommes heureux d&#039;ouvrir ce programme d&#039;acc\u00e8s anticip\u00e9 afin de permettre une mise \u00e0 disposition conforme du del-zota aux patients \u00e9ligibles dans les meilleurs d\u00e9lais. \u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Dans le cadre d&#039;un protocole de traitement autoris\u00e9 par le FDA, Avidity fournira le del-zota aux gar\u00e7ons et aux hommes atteints de DMD44 \u00e9ligibles, par l&#039;interm\u00e9diaire des professionnels de sant\u00e9 participants. Le recrutement devrait d\u00e9buter d&#039;ici la fin de l&#039;ann\u00e9e, et les participants \u00e0 l&#039;\u00e9tude EXPLORE44-OLE auront la possibilit\u00e9 de passer au protocole MAP apr\u00e8s deux ans de traitement.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>V\u00e9rifier maintenant<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/fr\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Mon fils est-il un candidat pour une th\u00e9rapie par saut d&#039;exon 44\u00a0?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Avidity s&#039;est associ\u00e9e \u00e0 FDA pour d\u00e9finir une strat\u00e9gie en vue du d\u00e9p\u00f4t d&#039;une demande d&#039;autorisation de mise sur le march\u00e9 (BLA) pour del-zota, suite \u00e0 une r\u00e9union pr\u00e9alable pr\u00e9vue en octobre 2025. Le d\u00e9p\u00f4t est programm\u00e9 pour 2026 afin d&#039;obtenir une approbation acc\u00e9l\u00e9r\u00e9e. Les participants passeront \u00e0 la commercialisation du m\u00e9dicament d\u00e8s l&#039;obtention de l&#039;approbation de FDA et la disponibilit\u00e9 du produit.<a href=\"https:\/\/investors.aviditybiosciences.com\/2025-11-19-Avidity-Biosciences-Announces-U-S-Managed-Access-Program-MAP-for-Investigational-Therapy-del-zota-in-DMD44#print\" target=\"_blank\" rel=\"noopener\"><\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Suivre cette page<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/fr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Tous les essais cliniques pour la dystrophie musculaire de Duchenne<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Avidity Biosciences announced its Managed Access Program (MAP) for investigational therapy delpacibart zotadirsen (del-zota) for eligible people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the United States. &#8220;We recognize the considerable needs facing the DMD44 community given there are no approved exon skipping therapies for this disease as well as [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5185,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[126,298,397,129,141,127,246],"class_list":["post-5183","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-avidity-biosciences","tag-del-zota","tag-delpacibart-zotadirsen","tag-dmd44","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies"],"subtitle":"Avidity Biosciences a lanc\u00e9 un programme d'acc\u00e8s g\u00e9r\u00e9 (MAP) aux \u00c9tats-Unis pour fournir le delpacibart zotadirsen exp\u00e9rimental (del-zota) aux patients atteints de mutations de la dystrophie musculaire de Duchenne (DMD) susceptibles de subir un saut de l'exon 44 (DMD44).","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/5183","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/comments?post=5183"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/5183\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/5185"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=5183"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=5183"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=5183"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}