{"id":7968,"date":"2026-05-14T14:59:27","date_gmt":"2026-05-14T11:59:27","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=7968"},"modified":"2026-05-14T15:47:11","modified_gmt":"2026-05-14T12:47:11","slug":"rgx-202-phase-3-affinity-duchenne-results-released-by-regenxbio","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/rgx-202-phase-3-affinity-duchenne-results-released-by-regenxbio\/","title":{"rendered":"RGX-202 Duchenne Gen Terapisi, NSAA \u0130yile\u015fmesi ve Mikrodistrofin Ekspresyonu G\u00f6steriyor \u2014 Ancak \u00d6nemli Biyobelirte\u00e7 Verileri Eksik"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>REGENXBIO k\u0131sa s\u00fcre \u00f6nce, Duchenne kas distrofisi (DMD) i\u00e7in deneysel bir gen terapisi olan RGX-202&#039;yi de\u011ferlendiren AFFINITY DUCHENNE \u00e7al\u0131\u015fmas\u0131n\u0131n Faz 3 a\u015famas\u0131ndan elde edilen olumlu sonu\u00e7lar\u0131 a\u00e7\u0131klad\u0131.<\/strong> \u015eirketin a\u00e7\u0131klamas\u0131na g\u00f6re, \u00e7al\u0131\u015fma y\u00fcksek istatistiksel anlaml\u0131l\u0131kla birincil hedefine ula\u015fm\u0131\u015f ve tedavi edilen hastalarda cesaret verici fonksiyonel iyile\u015fmeler de g\u00f6stermi\u015ftir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">AFFINITY DUCHENNE Denemesi Ne G\u00f6sterdi?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Bas\u0131n b\u00fcltenindeki en \u00f6nemli bulgu, hastalar\u0131n y\u00fczde 93'\u00fcn\u00fcn RGX-202 ile tedaviden 12 hafta sonra y\u00fczde 10'dan fazla mikrodistrofin ekspresyonuna ula\u015ft\u0131\u011f\u0131yd\u0131. Ara\u015ft\u0131rmac\u0131lar ayr\u0131ca kat\u0131l\u0131mc\u0131lar genelinde ortalama y\u00fczde 71.1'lik bir mikrodistrofin ekspresyon seviyesi bildirdi.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Birincil sonuca y\u00fcksek istatistiksel anlaml\u0131l\u0131kla ula\u015f\u0131ld\u0131; hastalar\u0131n y\u00fczde 93'\u00fcnde mikrodistrofin ekspresyonu y\u00fczde 10'un \u00fczerinde ger\u00e7ekle\u015fti (p&lt;0.0001).<\/strong><\/li>\n\n\n\n<li>RGX-202 mikrodistrofin ekspresyonu ile fonksiyonel iyile\u015fme aras\u0131nda istatistiksel olarak anlaml\u0131 bir korelasyon (NSAA n=9) bulunmu\u015ftur; bu da vekil u\u00e7 noktan\u0131n ge\u00e7erlili\u011fini desteklemektedir.<\/li>\n\n\n\n<li><strong>\u0130yi tolere edilen, farkl\u0131la\u015ft\u0131r\u0131lm\u0131\u015f g\u00fcvenlik profili<\/strong><\/li>\n\n\n\n<li>\u015eirket, 2027&#039;de h\u0131zland\u0131r\u0131lm\u0131\u015f onay alma olas\u0131l\u0131\u011f\u0131na haz\u0131rlan\u0131yor.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Ek olarak, ara d\u00f6nem fonksiyonel veriler, hastalar\u0131n NSAA (North Star Ambulatory Assessment) puanlar\u0131nda ve a\u015fa\u011f\u0131daki gibi zamanl\u0131 fonksiyonel testlerde iyile\u015fme ya\u015fad\u0131\u011f\u0131n\u0131 g\u00f6stermi\u015ftir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Aya\u011fa Kalkma Zaman\u0131<\/li>\n\n\n\n<li>10 Metre Y\u00fcr\u00fcy\u00fc\u015f\/Ko\u015fu<\/li>\n\n\n\n<li>T\u0131rmanma Zaman\u0131<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Bu sonu\u00e7lar \u00f6nemlidir \u00e7\u00fcnk\u00fc NSAA, Duchenne kas distrofisi olan erkek \u00e7ocuklarda motor fonksiyonu de\u011ferlendirmek i\u00e7in en yayg\u0131n kullan\u0131lan klinik ara\u00e7lardan biridir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">NSAA Geli\u015fimi Neden \u00d6nemli?<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">NSAA Ger\u00e7ek Hayattaki Fonksiyonel Yetenekleri \u00d6l\u00e7\u00fcyor<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">NSAA \u00f6l\u00e7e\u011fi, aya\u011fa kalkma, merdiven \u00e7\u0131kma, y\u00fcr\u00fcme ve z\u0131plama gibi g\u00fcnl\u00fck fiziksel yetenekleri de\u011ferlendirir. Duchenne kas distrofisinde, kas hasar\u0131 ilerledik\u00e7e NSAA puanlar\u0131 genellikle zamanla azal\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">NSAA skorlar\u0131nda stabilizasyon veya iyile\u015fme, tedavinin kas fonksiyonunu korumaya veya hastal\u0131\u011f\u0131n ilerlemesini yava\u015flatmaya yard\u0131mc\u0131 oldu\u011funu g\u00f6sterebilir.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Mikrodistrofin ve Fonksiyonel \u0130yile\u015fme Aras\u0131ndaki Korelasyon<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">REGENXBIO ayr\u0131ca RGX-202&#039;nin mikrodistrofin ekspresyonu ile bir y\u0131l sonraki fonksiyonel iyile\u015fme aras\u0131nda istatistiksel olarak anlaml\u0131 bir korelasyon g\u00f6sterdi\u011fini belirtti. Bu \u00f6zellikle \u00f6nemlidir \u00e7\u00fcnk\u00fc FDA gibi d\u00fczenleyiciler, biyobelirte\u00e7 iyile\u015fmelerinin hastalar i\u00e7in anlaml\u0131 klinik faydaya d\u00f6n\u00fc\u015ft\u00fc\u011f\u00fcne dair kan\u0131t g\u00f6rmek istemektedirler. Daha Fazla Bilgi Edinin: <a href=\"https:\/\/dmdwarrior.com\/tr\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" data-type=\"post\" data-id=\"4959\" target=\"_blank\" rel=\"noreferrer noopener\">NSAA<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Mikrodistrofin ekspresyonu g\u00f6zlemlendi.<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">Hastalar\u0131n Y\u00fczde 93'\u00fc  Y\u00fczde 10 E\u015fi\u011fini A\u015ft\u0131<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">En \u00f6nemli bulgulardan biri, kat\u0131l\u0131mc\u0131lar\u0131n y\u00fczde 93'\u00fcn\u00fcn mikrodistrofin ekspresyonunun y\u00fczde 10'un \u00fczerinde olmas\u0131yd\u0131. Duchenne kas distrofisi alan\u0131nda, bu e\u015fik genellikle klinik olarak anlaml\u0131 kabul edilir \u00e7\u00fcnk\u00fc daha y\u00fcksek distrofin seviyeleri kas stabilitesini art\u0131rabilir ve zaman i\u00e7inde dejenerasyonu azaltabilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket ayr\u0131ca hastalar\u0131n y\u00fczde 80&#039;inde mikrodistrofin ekspresyonunun y\u00fczde 40&#039;\u0131n \u00fczerine \u00e7\u0131kt\u0131\u011f\u0131n\u0131 bildirdi; bu da DMD&#039;de gen terapisi \u00e7al\u0131\u015fmas\u0131 i\u00e7in olduk\u00e7a y\u00fcksek bir seviye olarak kabul ediliyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha Fazlas\u0131n\u0131 Bul: <a href=\"https:\/\/regenxbio.gcs-web.com\/static-files\/b554d29e-1bb8-40f1-ba4c-e1494d5172f1\" target=\"_blank\" rel=\"noreferrer noopener\">AFFINITY DUCHENNE RGX-202&#039;nin Temel \u00c7al\u0131\u015fmas\u0131<\/a><\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Ancak, \u00d6nemli Biyobelirte\u00e7 Verileri Payla\u015f\u0131lmad\u0131<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">Kreatin Kinaz (CK), AST ve ALT Verileri A\u00e7\u0131klanmad\u0131<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Umut vadeden genel sonu\u00e7lara ra\u011fmen, bas\u0131n b\u00fclteninde a\u015fa\u011f\u0131dakiler de dahil olmak \u00fczere birka\u00e7 kritik biyobelirte\u00e7 hakk\u0131nda ayr\u0131nt\u0131l\u0131 veriler yer almad\u0131:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><a href=\"https:\/\/dmdwarrior.com\/tr\/what-is-creatine-kinase-ck\/\" target=\"_blank\" data-type=\"post\" data-id=\"2167\" rel=\"noreferrer noopener\">Kreatin Kinaz (CK)<\/a><\/li>\n\n\n\n<li>Aspartat Aminotransferaz (AST)<\/li>\n\n\n\n<li>Alanin Aminotransferaz (ALT)<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Bu eksiklik \u00f6nemlidir \u00e7\u00fcnk\u00fc bu biyobelirte\u00e7ler, Duchenne kas distrofisinde kas hasar\u0131n\u0131 ve karaci\u011ferle ilgili g\u00fcvenlik endi\u015felerini de\u011ferlendirmek i\u00e7in yayg\u0131n olarak kullan\u0131lmaktad\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Duchenne Kas Distrofisinde CK, AST ve ALT Neden Bu Kadar \u00d6nemli?<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">Kreatin Kinaz (CK)<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">CK, Duchenne kas distrofisinde en \u00f6nemli biyobelirte\u00e7lerden biridir. Y\u00fcksek CK seviyeleri, devam eden kas y\u0131k\u0131m\u0131n\u0131 g\u00f6sterir. E\u011fer gen terapisi ger\u00e7ekten kas liflerini koruyorsa, bir\u00e7ok uzman CK seviyelerinin zamanla azalmas\u0131n\u0131 bekler.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">CK verileri olmadan, tedavinin biyokimyasal d\u00fczeyde aktif kas hasar\u0131n\u0131 azalt\u0131p azaltmad\u0131\u011f\u0131n\u0131 anlamak daha zor hale gelir.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">AST ve ALT<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">AST ve ALT genellikle karaci\u011fer enzimleri olarak bilinir, ancak Duchenne hastalar\u0131nda kas hasar\u0131 nedeniyle de y\u00fckselebilirler. Y\u00fcksek AST ve ALT seviyeleri \u015funlar\u0131 yans\u0131tabilir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>S\u00fcregelen kas y\u0131k\u0131m\u0131<\/li>\n\n\n\n<li>Karaci\u011fer iltihab\u0131<\/li>\n\n\n\n<li>Potansiyel tedaviyle ili\u015fkili toksisite<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Bu biyobelirte\u00e7lerin takibi, \u00f6zellikle gen terapisi denemelerinde b\u00fcy\u00fck \u00f6nem ta\u015f\u0131maktad\u0131r \u00e7\u00fcnk\u00fc karaci\u011ferle ilgili yan etkiler bu alanda \u00f6nemli bir g\u00fcvenlik endi\u015fesi olmaya devam etmektedir. Daha fazla bilgi i\u00e7in: <a href=\"https:\/\/dmdwarrior.com\/tr\/ck-levels-after-duchenne-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">CK, AST ve ALT Verilerinin \u00d6nemi<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Biyobelirte\u00e7 \u015eeffafl\u0131\u011f\u0131 Neden \u00d6nemlidir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Mikrodistrofin ekspresyonu ve NSAA&#039;daki iyile\u015fme cesaret verici bulgular olsa da, CK, AST ve ALT gibi biyobelirte\u00e7ler, tedavinin v\u00fccudu biyolojik olarak nas\u0131l etkiledi\u011fine dair bir ba\u015fka kritik kan\u0131t katman\u0131 sa\u011flamaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ailelerin, klinisyenlerin ve ara\u015ft\u0131rmac\u0131lar\u0131n daha iyi anlamalar\u0131 i\u00e7in \u015feffaf biyobelirte\u00e7 verilerine ihtiya\u00e7lar\u0131 var:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Kas hasar\u0131n\u0131n ger\u00e7ekten azal\u0131p azalmad\u0131\u011f\u0131<\/li>\n\n\n\n<li>Tedavi etkisinin ne kadar kal\u0131c\u0131 olabilece\u011fi<\/li>\n\n\n\n<li>G\u00fcvenlik endi\u015felerinin zaman i\u00e7inde ortaya \u00e7\u0131k\u0131p \u00e7\u0131kmad\u0131\u011f\u0131<\/li>\n\n\n\n<li>RGX-202&#039;nin di\u011fer Duchenne gen terapileriyle kar\u015f\u0131la\u015ft\u0131r\u0131lmas\u0131<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne kas distrofisi toplulu\u011fu, anlaml\u0131 tedaviler i\u00e7in on y\u0131llard\u0131r bekliyor. Olumlu fonksiyonel veriler ve g\u00fc\u00e7l\u00fc mikrodistrofin ekspresyonu cesaret verici ad\u0131mlar olsa da, RGX-202&#039;nin uzun vadeli klinik de\u011ferini ve g\u00fcvenlik profilini tam olarak de\u011ferlendirmek i\u00e7in kapsaml\u0131 biyobelirte\u00e7 \u015feffafl\u0131\u011f\u0131 \u015fart olacakt\u0131r.<\/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>REGENXBIO recently announced positive topline results from the Phase 3 portion of the AFFINITY DUCHENNE trial evaluating RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD). According to the company, the study met its primary endpoint with high statistical significance, while also showing encouraging functional improvements in treated patients. What Did the AFFINITY DUCHENNE [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":7969,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[525,137,61,344,107,108],"class_list":["post-7968","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-affinity-duchenne","tag-clinical-trial","tag-gene-therapy","tag-microdystrophin","tag-regenxbio","tag-rgx-202"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7968","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=7968"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7968\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/7969"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=7968"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=7968"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=7968"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}