{"id":4274,"date":"2025-08-04T17:03:02","date_gmt":"2025-08-04T14:03:02","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4274"},"modified":"2025-08-04T17:13:24","modified_gmt":"2025-08-04T14:13:24","slug":"dyne-therapeutics-announces-fda-breakthrough-therapy-designation-for-dyne-251-in-duchenne-muscular-dystrophy-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/dyne-therapeutics-announces-fda-breakthrough-therapy-designation-for-dyne-251-in-duchenne-muscular-dystrophy-dmd\/","title":{"rendered":"Dyne Therapeutics, Duchenne Musk\u00fcler Distrofi (DMD) Tedavisinde DYNE-251 i\u00e7in FDA \u00c7\u0131\u011f\u0131r A\u00e7an Terapi Tan\u0131m\u0131n\u0131 Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Dyne Therapeutics, ABD G\u0131da ve \u0130la\u00e7 Dairesi'nin (FDA), DYNE-251'ye, ekzon 51 atlamas\u0131na uygun Duchenne kas distrofisi (DMD) hastalar\u0131n\u0131n tedavisi i\u00e7in \u00c7\u0131\u011f\u0131r A\u00e7an Tedavi Tan\u0131m\u0131 verdi\u011fini duyurdu. Tan\u0131m, devam eden DELIVER klinik \u00e7al\u0131\u015fmas\u0131ndan elde edilen verilere dayanmaktad\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">DYNE-251: \u00c7\u0131\u011f\u0131r A\u00e7an Terapi Tan\u0131m\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Dyne&#039;\u0131n ba\u015f t\u0131p sorumlusu Dr. Doug Kerr, &quot;DYNE-251 i\u00e7in bu \u00c7\u0131\u011f\u0131r A\u00e7an Terapi Unvan\u0131, ekzon 51 atlaman\u0131n neredeyse tam uzunlukta distrofin \u00fcretimine yol a\u00e7abilece\u011fi DMD&#039;li bireylerde anlaml\u0131 i\u015flevsel iyile\u015fme sa\u011flamak \u00fczere tasarlanm\u0131\u015f yeni nesil bir tedavi olarak potansiyelinin bir kan\u0131t\u0131d\u0131r,&quot; dedi. &quot;Daha \u00f6nce de a\u00e7\u0131klad\u0131\u011f\u0131m\u0131z gibi, DYNE-251, aya\u011fa kalkma s\u00fcresi ve 95. y\u00fczdelik ad\u0131m h\u0131z\u0131 gibi temel \u00f6l\u00e7\u00fctlerle de\u011ferlendirildi\u011finde, on sekiz ay boyunca s\u00fcrekli i\u015flevsel iyile\u015fme g\u00f6stermi\u015ftir. G\u00f6zlemlenen neredeyse tam uzunlukta distrofin ekspresyonu seviyesi, hastalara potansiyel olarak anlaml\u0131 faydalar sa\u011flama yolunda \u00f6nemli bir ad\u0131md\u0131r.&quot;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Musk\u00fcler Distrofi Tedavisinde Yakla\u015fan Ekzon 51 Atlama Terapileri<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/dyne-therapeutics-receives-european-medicines-agency-ema-orphan-drug-designation-for-dyne-251-in-duchenne-muscular-dystrophy\/\">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<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-251 for the treatment of patients with Duchenne muscular dystrophy (DMD), amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER clinical trial. DYNE-251: Breakthrough Therapy Designation \u201cThis Breakthrough Therapy Designation for DYNE-251 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4275,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[208,145,122,193,194,88],"class_list":["post-4274","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-fda"],"subtitle":"Duchenne kas distrofisi (DMD) hastalar\u0131nda 51. ekzonu atlayabilenleri tedavi etmek i\u00e7in Dyne Therapeutics, FDA'nin DYNE-251'ye \u00c7\u0131\u011f\u0131r A\u00e7an Tedavi Onay\u0131 verdi\u011fini duyurdu. Bu onay, devam eden DELIVER klinik \u00e7al\u0131\u015fmas\u0131ndan elde edildi.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4274","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=4274"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4274\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4275"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4274"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4274"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4274"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}