{"id":3758,"date":"2025-04-24T14:48:38","date_gmt":"2025-04-24T11:48:38","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3758"},"modified":"2025-04-24T14:57:12","modified_gmt":"2025-04-24T11:57:12","slug":"dyne-therapeutics-receives-european-medicines-agency-ema-orphan-drug-designation-for-dyne-251-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/dyne-therapeutics-receives-european-medicines-agency-ema-orphan-drug-designation-for-dyne-251-in-duchenne-muscular-dystrophy\/","title":{"rendered":"Dyne Therapeutics, Duchenne Musk\u00fcler Distrofisi&#039;nde DYNE-251 i\u00e7in Avrupa \u0130la\u00e7 Ajans\u0131 (EMA) Yetim \u0130la\u00e7 Tan\u0131m\u0131n\u0131 Ald\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Genetik olarak y\u00f6nlendirilen n\u00f6rom\u00fcsk\u00fcler hastal\u0131klar\u0131 olan bireyler i\u00e7in hayat de\u011fi\u015ftiren tedaviler geli\u015ftirmeye adanm\u0131\u015f klinik a\u015famadaki bir \u015firket olan Dyne Therapeutics, DYNE-251&#039;nin Duchenne kas distrofisi (DMD) tedavisi i\u00e7in Avrupa Komisyonu (EC) taraf\u0131ndan yetim ila\u00e7 olarak belirlendi\u011fini duyurdu. DYNE-251, Faz 1\/2 DELIVER k\u00fcresel klinik \u00e7al\u0131\u015fmas\u0131nda ekzon 51 atlamas\u0131na duyarl\u0131 olan DMD hastalar\u0131nda test ediliyor.<a href=\"https:\/\/dmdwarrior.com\/tr\/exon-51-skipping-therapies-dmd\/\" data-type=\"post\" data-id=\"3429\">Ekzon 51 Atlama Terapileri<\/a>)<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0130\u00e7indekiler<\/h2><nav><ul><li><a href=\"#phase-1-2-trial-of-dyne-251\">DYNE-251&#039;nin 1\/2 faz denemesi<\/a><\/li><li><a href=\"#what-is-orphan-drug\">Yetim ila\u00e7 nedir?<\/a><\/li><li><a href=\"#key-milestones-for-the-deliver-trial\">DELIVER Denemesinin \u00d6nemli Kilometre Ta\u015flar\u0131<\/a><\/li><li><a href=\"#about-dyne-251\">DYNE-251 Hakk\u0131nda<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"phase-1-2-trial-of-dyne-251\">DYNE-251&#039;nin 1\/2 faz denemesi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Mart ay\u0131ndaki 2025 Muscular Dystrophy Association (MDA) Klinik ve Bilimsel Konferans\u0131&#039;nda, DYNE-251&#039;nin devam eden DELIVER \u00e7al\u0131\u015fmas\u0131ndan uzun vadeli klinik veriler sunuldu. Bu veriler, se\u00e7ilen kay\u0131t dozunda benzersiz ve s\u00fcrd\u00fcr\u00fclebilir bir i\u015flevsel iyile\u015fme g\u00f6sterdi. Avrupa&#039;daki DMD klinik \u00e7al\u0131\u015fmalar\u0131 i\u00e7in birincil bir son nokta olarak kabul edilen nesnel bir dijital sonu\u00e7 olan Stride Velocity 95th Centile (SV95C), DELIVER \u00e7al\u0131\u015fmas\u0131nda kullan\u0131lan i\u015flevsel de\u011ferlendirmelerden biridir. (Daha fazlas\u0131n\u0131 okuyun: <a href=\"https:\/\/dmdwarrior.com\/tr\/dyne-therapeutics-announces-phase-1-2-deliver-trial-of-dyne-251-in-duchenne-muscular-dystrophy\/\">A\u015fama 1\/2 DELIVER DYNE-251 Denemesi<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-orphan-drug\">Yetim ila\u00e7 nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">AB, Avrupa Birli\u011fi&#039;nde (AB) 10.000 ki\u015fiden be\u015finden az\u0131n\u0131 etkileyen nadir, ya\u015fam\u0131 tehdit eden veya kronik olarak zay\u0131flat\u0131c\u0131 hastal\u0131klar\u0131n veya durumlar\u0131n tedavisi, te\u015fhisi veya \u00f6nlenmesi i\u00e7in tasarlanm\u0131\u015f ila\u00e7lara ve biyolojiklere yetim ila\u00e7 stat\u00fcs\u00fc verir. Yetim stat\u00fcs\u00fc, \u015firketlere azalt\u0131lm\u0131\u015f d\u00fczenleyici \u00fccretler, klinik protokol yard\u0131m\u0131, ara\u015ft\u0131rma hibeleri ve onaylan\u0131rsa AB&#039;de 10 y\u0131la kadar pazar m\u00fcnhas\u0131rl\u0131\u011f\u0131 potansiyeli gibi belirli avantajlar sa\u011flar. DYNE-251 ayr\u0131ca Mart 2023&#039;te ABD G\u0131da ve \u0130la\u00e7 Dairesi (FDA) yetim ila\u00e7 ve nadir pediatrik hastal\u0131k stat\u00fclerini ald\u0131.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"key-milestones-for-the-deliver-trial\">DELIVER Denemesinin \u00d6nemli Kilometre Ta\u015flar\u0131<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Dyne, ekzon 51 atlamaya uygun DMD hastalar\u0131nda DYNE-251 i\u00e7in d\u00fcnya \u00e7ap\u0131nda h\u0131zland\u0131r\u0131lm\u0131\u015f onay yollar\u0131n\u0131 izlemeye devam ediyor.<\/li>\n\n\n\n<li>Dyne, DELIVER denemesinin bir par\u00e7as\u0131 olarak 32 hastadan olu\u015fan kay\u0131t geni\u015fletme kohortunu tamamen kaydetti. Bu kohorttan elde edilen verilerin 2025 sonu i\u00e7in planlanmas\u0131 planlan\u0131yor.<\/li>\n\n\n\n<li>Dyne, 2026 y\u0131l\u0131 ba\u015flar\u0131nda ABD&#039;de h\u0131zland\u0131r\u0131lm\u0131\u015f onay i\u00e7in Biyolojik Lisans Ba\u015fvurusu (BLA) ba\u015fvurusunda bulunmay\u0131 planl\u0131yor.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"about-dyne-251\">DYNE-251 Hakk\u0131nda<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DYNE-251, ekzon 51 atlamaya uygun DMD&#039;li ki\u015filer i\u00e7in Faz 1\/2 global DELIVER klinik \u00e7al\u0131\u015fmas\u0131nda de\u011ferlendirilen deneysel bir tedavidir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DYNE-251, ABD G\u0131da ve \u0130la\u00e7 Dairesi taraf\u0131ndan ekzon 51 atlamaya uygun DMD mutasyonlar\u0131n\u0131n tedavisi i\u00e7in h\u0131zl\u0131 tedavi, yetim ila\u00e7 ve nadir pediatrik hastal\u0131k tan\u0131mlar\u0131na lay\u0131k g\u00f6r\u00fcld\u00fc.<\/p>","protected":false},"excerpt":{"rendered":"<p>Today, Dyne Therapeutics, a clinical-stage company dedicated to developing life-changing treatments for individuals with genetically driven neuromuscular diseases, announced that DYNE-251 has been designated as an orphan drug by the European Commission (EC) for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being tested in DMD patients who are susceptible to exon 51 skipping [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3760,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[143,208,145,91,213,122,193,194,195],"class_list":["post-3758","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne","tag-dyne-therapeutics","tag-dyne-251","tag-ema","tag-european-medicines-agency","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-orphan-drug"],"subtitle":"Dyne Therapeutics, bug\u00fcn Avrupa Komisyonu'nun (EC) DYNE-251'ye Duchenne kas distrofisi (DMD) tedavisi i\u00e7in yetim ila\u00e7 stat\u00fcs\u00fc verdi\u011fini duyurdu.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3758","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=3758"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3758\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3760"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3758"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3758"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3758"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}