{"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\/tr\/avidity-biosciences-announces-u-s-managed-access-program-map-for-investigational-therapy-del-zota-in-dmd44\/","title":{"rendered":"Avidity Biosciences, Ara\u015ft\u0131rma Terapisi del-zota (Ekzon 44 Atlama) i\u00e7in ABD Y\u00f6netilen Eri\u015fim Program\u0131n\u0131 (MAP) Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Avidity Biosciences, ABD&#039;de ekzon 44 atlamaya (DMD44) uygun Duchenne kas distrofisi mutasyonlar\u0131na sahip uygun ki\u015filer i\u00e7in ara\u015ft\u0131rma tedavisi delpacibart zotadirsen (del-zota) i\u00e7in Y\u00f6netilen Eri\u015fim Program\u0131n\u0131 (MAP) duyurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cAvidity Ba\u015fkan\u0131 ve \u0130cra Kurulu Ba\u015fkan\u0131 Sarah Boyce, &quot;Bu hastal\u0131k i\u00e7in onayl\u0131 ekzon atlama tedavilerinin olmamas\u0131 ve klinik \u00e7al\u0131\u015fmalar\u0131m\u0131zda g\u00f6sterilen benzersiz verilere dayanarak del-zota&#039;nin potansiyeli g\u00f6z \u00f6n\u00fcne al\u0131nd\u0131\u011f\u0131nda, DMD44 toplulu\u011funun kar\u015f\u0131 kar\u015f\u0131ya oldu\u011fu \u00f6nemli ihtiya\u00e7lar\u0131n fark\u0131nday\u0131z,&quot; dedi. &quot;Uygun hastalara m\u00fcmk\u00fcn olan en k\u0131sa s\u00fcrede del-zota sa\u011flamak i\u00e7in uyumlu bir yakla\u015f\u0131m sa\u011flamak amac\u0131yla bu MAP&#039;\u0131 a\u00e7maktan mutluluk duyuyoruz.&quot;\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">FDA onayl\u0131 bir tedavi protokol\u00fc kapsam\u0131nda Avidity, kat\u0131l\u0131mc\u0131 sa\u011fl\u0131k hizmeti sa\u011flay\u0131c\u0131lar\u0131 arac\u0131l\u0131\u011f\u0131yla DMD44&#039;l\u00fc uygun erkek ve erkek \u00e7ocuklar\u0131na del-zota sa\u011flayacakt\u0131r. Kay\u0131tlar\u0131n y\u0131l sonuna kadar ba\u015flamas\u0131 bekleniyor ve EXPLORE44-OLE kat\u0131l\u0131mc\u0131lar\u0131, 2 y\u0131ll\u0131k tedaviyi tamamlad\u0131ktan sonra MAP&#039;ye ge\u00e7i\u015f yapma se\u00e7ene\u011fine sahip olacaklar.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u015eimdi Kontrol Edin<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">O\u011flum ekzon 44 atlama terapisi i\u00e7in uygun bir aday m\u0131d\u0131r?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Avidity, Ekim 2025&#039;te yap\u0131lan BLA \u00f6ncesi toplant\u0131n\u0131n ard\u0131ndan del-zota i\u00e7in BLA ba\u015fvurusunda bulunmak \u00fczere FDA ile ayn\u0131 yolda ilerlemektedir ve ba\u015fvurunun h\u0131zland\u0131r\u0131lm\u0131\u015f onay i\u00e7in 2026&#039;da ger\u00e7ekle\u015ftirilmesi planlanmaktad\u0131r. Kat\u0131l\u0131mc\u0131lar, gelecekteki olas\u0131 FDA onay\u0131 ve \u00fcr\u00fcn bulunabilirli\u011fi sonras\u0131nda ticari ila\u00e7 tedarikine ge\u00e7ecektir.<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>Bu Sayfay\u0131 Takip Edin<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne i\u00e7in T\u00fcm Klinik Ara\u015ft\u0131rmalar<\/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, ekzon 44 atlamaya (DMD44) uygun Duchenne kas distrofisi (DMD) mutasyonlar\u0131 olan hastalara ara\u015ft\u0131rma a\u015famas\u0131ndaki delpacibart zotadirsen (del-zota) sa\u011flamak i\u00e7in ABD'de bir Y\u00f6netilen Eri\u015fim Program\u0131 (MAP) ba\u015flatt\u0131.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5183","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=5183"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5183\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5185"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5183"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5183"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5183"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}