{"id":5310,"date":"2025-12-11T16:56:58","date_gmt":"2025-12-11T13:56:58","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5310"},"modified":"2025-12-11T16:57:00","modified_gmt":"2025-12-11T13:57:00","slug":"atossa-therapeutics-receives-fda-rare-pediatric-disease-designation-for-z-endoxifen-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/atossa-therapeutics-receives-fda-rare-pediatric-disease-designation-for-z-endoxifen-for-duchenne-muscular-dystrophy\/","title":{"rendered":"Atossa Therapeutics, Duchenne Musk\u00fcler Distrofisi i\u00e7in (Z)-Endoksifen'e FDA Nadir Pediatrik Hastal\u0131k Tan\u0131m\u0131 Ald\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Atossa Therapeutics, ABD G\u0131da ve \u0130la\u00e7 \u0130daresi&#039;nin (\u201cFDA\u201d) Duchenne Musk\u00fcler Distrofisi (\u201cDMD\u201d) tedavisi i\u00e7in (Z)-Endoxifen&#039;ye Nadir Pediatrik Hastal\u0131k (\u201cRPD\u201d) stat\u00fcs\u00fc verdi\u011fini duyurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cAtossa Therapeutics Ba\u015fkan\u0131 ve CEO&#039;su Steven Quay, MD, Ph.D., \u201dBu onay, Atossa i\u00e7in \u00f6nemli bir d\u00fczenleyici d\u00f6n\u00fcm noktas\u0131d\u0131r ve (Z)-Endoxifen&#039;nin Duchenne Musk\u00fcler Distrofisi tedavisi olarak potansiyelini destekleyen bilimin g\u00fc\u00e7l\u00fc bir do\u011frulamas\u0131 oldu\u011funa inan\u0131yoruz\u201c dedi. \u201dDMD, en y\u0131k\u0131c\u0131 \u00e7ocukluk \u00e7a\u011f\u0131 hastal\u0131klar\u0131ndan biridir. Ailelerin steroidler ve gen hedefli yakla\u015f\u0131mlar\u0131n \u00f6tesinde daha iyi se\u00e7eneklere acilen ihtiyac\u0131 var. Onkoloji temel odak noktam\u0131z olmaya devam ederken, bu d\u00f6n\u00fcm noktas\u0131 (Z)-Endoxifen&#039;nin hem kanser hem de nadir hastal\u0131klarda bir platform tedavisi olarak potansiyelini vurgulayarak, Nadir Pediatrik Hastal\u0131klar program\u0131 arac\u0131l\u0131\u011f\u0131yla seyreltici olmayan de\u011fer yaratman\u0131n kap\u0131s\u0131n\u0131 a\u00e7\u0131yor.\u201d<\/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=\"#what-is-endoxifen\">Endoxifen nedir?<\/a><\/li><li><a href=\"#why-endoxifen-in-dmd\">DMD&#039;de Endoxifen neden kullan\u0131l\u0131r?<\/a><\/li><li><a href=\"#unmet-need-and-treatment-landscape\">Kar\u015f\u0131lanmayan \u0130htiya\u00e7 ve Tedavi Ortam\u0131<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-endoxifen\">Endoxifen nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Endoxifen, deneysel bir k\u00fc\u00e7\u00fck molek\u00fcll\u00fc terapi olup, di\u011fer endikasyonlarda yayg\u0131n olarak kullan\u0131lan se\u00e7ici \u00f6strojen resept\u00f6r mod\u00fclat\u00f6r\u00fc (SERM) tamoksifenin ba\u015fl\u0131ca aktif metabolitidir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Endoksifen, \u00f6strojen resept\u00f6rleriyle do\u011frudan etkile\u015fime girdi\u011fi i\u00e7in, kas b\u00fcy\u00fcmesi, onar\u0131m\u0131 ve fibrozis de dahil olmak \u00fczere kas sa\u011fl\u0131\u011f\u0131yla ilgili yollar\u0131 d\u00fczenleme potansiyeline sahiptir. Atossa, devam eden geli\u015ftirme ve de\u011ferlendirmeye tabi olarak, tutarl\u0131 ila\u00e7 maruziyetini sa\u011flamak ve uygun, g\u00fcnde bir kez dozlamay\u0131 m\u00fcmk\u00fcn k\u0131lmak \u00fczere tasarlanm\u0131\u015f oral bir endoksifen form\u00fclasyonu geli\u015ftirmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Endoxifen, DMD veya herhangi bir kas distrofisi endikasyonunda kullan\u0131m i\u00e7in onaylanmam\u0131\u015ft\u0131r. Bu kullan\u0131mlar i\u00e7in g\u00fcvenli\u011fi ve etkinli\u011fi kan\u0131tlanmam\u0131\u015ft\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"why-endoxifen-in-dmd\">DMD&#039;de Endoxifen neden kullan\u0131l\u0131r?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0130skelet kas\u0131, \u00f6strojen resept\u00f6r yollar\u0131 da dahil olmak \u00fczere hormonal sinyallere duyarl\u0131d\u0131r ve bu sinyaller \u015funlar\u0131 etkileyebilir:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Kas yenilenmesi ve onar\u0131m\u0131<\/li>\n\n\n\n<li>\u0130ltihaplanma ve oksidatif stres<\/li>\n\n\n\n<li>Fibrozis ve doku yeniden yap\u0131lanmas\u0131<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Se\u00e7ici \u00f6strojen resept\u00f6r mod\u00fclat\u00f6rleri (SERM&#039;ler) kaslarda biyolojik aktivite g\u00f6stermi\u015f olup \u00e7e\u015fitli n\u00f6rom\u00fcsk\u00fcler ba\u011flamlarda ara\u015ft\u0131r\u0131lmaktad\u0131r. <strong>G\u00fc\u00e7l\u00fc ve aktif bir metabolit olan endoksifen \u015funlar\u0131 sunabilir:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Kas dokusunda do\u011frudan \u00f6strojen resept\u00f6r\u00fc mod\u00fclasyonu<\/li>\n\n\n\n<li>A\u011f\u0131zdan dozlama potansiyeli, nihayetinde g\u00fcvenli ve etkili oldu\u011fu kan\u0131tlan\u0131rsa kronik uygulamay\u0131 destekleyebilir.<\/li>\n\n\n\n<li>Mutasyondan ba\u011f\u0131ms\u0131z bir mekanizma; yani prensip olarak, altta yatan distrofin geni mutasyonundan ba\u011f\u0131ms\u0131z olarak, daha geni\u015f DMD pop\u00fclasyonunda incelenebilir.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Atossa program\u0131, bu bilimsel gerek\u00e7eyi preklinik ve uygun g\u00f6r\u00fclmesi halinde gelecekteki klinik \u00e7al\u0131\u015fmalar yoluyla titizlikle de\u011ferlendirmek \u00fczere tasarlanm\u0131\u015ft\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"unmet-need-and-treatment-landscape\">Kar\u015f\u0131lanmayan \u0130htiya\u00e7 ve Tedavi Ortam\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">G\u00fcn\u00fcm\u00fczdeki DMD tedavisi genellikle \u015funlar\u0131 i\u00e7ermektedir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Kortikosteroidler, hastal\u0131\u011f\u0131n ilerlemesini yava\u015flatmaya yard\u0131mc\u0131 olabilir ancak \u00f6nemli yan etkilerle ili\u015fkilidir.<\/li>\n\n\n\n<li>Ekson atlama veya gen hedefli yakla\u015f\u0131mlar gibi mutasyona \u00f6zg\u00fc tedaviler, yaln\u0131zca belirli genetik varyantlara sahip hastalara uygulanabilir.<\/li>\n\n\n\n<li>Destekleyici bak\u0131m; kalp ve solunum y\u00f6netimi, fizyoterapi ve ortopedi hizmetlerini i\u00e7erir.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Mevcut tedavilerle birlikte veya tek ba\u015f\u0131na kullan\u0131labilecek, genel olarak uygulanabilir ek tedavi se\u00e7eneklerine a\u00e7\u0131k bir ihtiya\u00e7 vard\u0131r. Mutasyondan ba\u011f\u0131ms\u0131z, a\u011f\u0131zdan uygulanan bir tedavi \u2013e\u011fer g\u00fcvenli ve etkili oldu\u011fu kan\u0131tlan\u0131rsa\u2013 potansiyel olarak DMD&#039;li geni\u015f bir birey yelpazesine ula\u015fabilir.<\/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>Atossa Therapeutics announced that the U.S. Food and Drug Administration (&#8220;FDA&#8221;) has granted Rare Pediatric Disease (&#8220;RPD&#8221;) designation to (Z)-Endoxifen for the treatment of Duchenne Muscular Dystrophy (&#8220;DMD&#8221;). &#8220;This designation is an important regulatory milestone for Atossa, and we believe a strong validation of the science supporting the potential of (Z)-Endoxifen as a treatment for [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5312,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[461,462,464,463],"class_list":["post-5310","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-z-endoxifen","tag-atossa-therapeutics","tag-endoxifen","tag-tamoxifen"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5310","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=5310"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5310\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5312"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5310"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5310"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5310"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}