{"id":4357,"date":"2025-08-20T17:19:30","date_gmt":"2025-08-20T14:19:30","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4357"},"modified":"2025-08-20T17:21:43","modified_gmt":"2025-08-20T14:21:43","slug":"keros-announces-u-s-fda-orphan-drug-designation-granted-to-ker-065-for-the-treatment-of-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/keros-announces-u-s-fda-orphan-drug-designation-granted-to-ker-065-for-the-treatment-of-duchenne-muscular-dystrophy\/","title":{"rendered":"Keros, Duchenne Musk\u00fcler Distrofi Tedavisi i\u00e7in KER-065&#039;ye ABD FDA Yetim \u0130la\u00e7 Tan\u0131m\u0131n\u0131n Verildi\u011fini Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Keros Therapeutics bug\u00fcn ABD G\u0131da ve \u0130la\u00e7 Dairesi&#039;nin (&quot;FDA&quot;) KER-065&#039;ye Duchenne kas distrofisi (&quot;DMD&quot;) tedavisi i\u00e7in Yetim \u0130la\u00e7 stat\u00fcs\u00fc verdi\u011fini duyurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ba\u015fkan ve \u0130cra Kurulu Ba\u015fkan\u0131 Jasbir S. Seehra, &quot;KER-065 i\u00e7in Yetim \u0130la\u00e7 unvan\u0131n\u0131 almak, DMD hastalar\u0131 i\u00e7in kar\u015f\u0131lanmam\u0131\u015f \u00f6nemli t\u0131bbi ihtiyac\u0131 vurguluyor,&quot; dedi. &quot;Bu unvan, KER-065&#039;yi DMD hastalar\u0131nda Faz 2 klinik denemesine ilerletmemizde Keros i\u00e7in \u00f6nemli bir kilometre ta\u015f\u0131 g\u00f6revi g\u00f6r\u00fcyor.&quot;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">FDA, Amerika Birle\u015fik Devletleri&#039;nde 200.000&#039;den az ki\u015fiyi etkileyen nadir t\u0131bbi hastal\u0131klar veya rahats\u0131zl\u0131klar\u0131 ele alan ara\u015ft\u0131rma a\u015famas\u0131ndaki tedavilere Yetim \u0130la\u00e7 stat\u00fcs\u00fc vermektedir. Bu stat\u00fc, nitelikli klinik testler i\u00e7in vergi indirimleri, FDA ba\u015fvuru \u00fccretlerinin muafiyeti veya k\u0131smi \u00f6demesi ve onaylanmas\u0131 halinde yedi y\u0131ll\u0131k pazar m\u00fcnhas\u0131rl\u0131\u011f\u0131 gibi belirli potansiyel avantajlar sa\u011flamaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/keros-therapeutics-announces-initial-topline-results-from-the-phase-1-clinical-trial-of-ker-065-in-healthy-volunteers\/\" target=\"_blank\" rel=\"noreferrer noopener\">Sa\u011fl\u0131kl\u0131 G\u00f6n\u00fcll\u00fclerde KER-065&#039;nin 1. Faz Klinik \u00c7al\u0131\u015fmas\u0131<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">KER-065 nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">KER-065, aktivin resept\u00f6r\u00fc tip IIA ve aktivin resept\u00f6r\u00fc tip IIB&#039;den t\u00fcretilen ve Fc alan\u0131 olarak bilinen insan antikorunun bir k\u0131sm\u0131na kayna\u015ft\u0131r\u0131lm\u0131\u015f modifiye edilmi\u015f bir ligand ba\u011flanma alan\u0131ndan olu\u015fan yeni bir ligand tuza\u011f\u0131d\u0131r. KER-065, bir ligand tuza\u011f\u0131 g\u00f6revi g\u00f6recek ve aktivin resept\u00f6rleri arac\u0131l\u0131\u011f\u0131yla sinyal g\u00f6nderen iki ligand olan miyostatin ve aktivin A&#039;n\u0131n biyolojik etkilerini inhibe edecek \u015fekilde tasarlanm\u0131\u015ft\u0131r. Bu ligandlar, iskelet kas\u0131 rejenerasyonunu, kas boyutunu ve g\u00fcc\u00fcn\u00fc art\u0131r\u0131r, v\u00fccut ya\u011f\u0131n\u0131 azalt\u0131r, iskelet kas\u0131 fibrozunu azalt\u0131r ve kemik g\u00fcc\u00fcn\u00fc art\u0131r\u0131r.<\/p>","protected":false},"excerpt":{"rendered":"<p>Keros Therapeutics today announced the U.S. Food and Drug Administration (\u201cFDA\u201d) granted Orphan Drug designation for KER-065 for the treatment of Duchenne muscular dystrophy (\u201cDMD\u201d). \u201cReceiving Orphan Drug designation for KER-065 highlights the significant unmet medical need for patients with DMD,\u201d said Jasbir S. Seehra, President and Chief Executive Officer. \u201cThis designation serves as a [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4360,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[337,336,195],"class_list":["post-4357","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-ker-065","tag-keros-therapeutics","tag-orphan-drug"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4357","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=4357"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4357\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4360"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4357"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4357"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4357"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}