{"id":6289,"date":"2026-04-29T17:14:16","date_gmt":"2026-04-29T14:14:16","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6289"},"modified":"2026-04-29T17:16:44","modified_gmt":"2026-04-29T14:16:44","slug":"precision-biosciences-pbgene-dmd-phase-1-2a-function-dmd-clinical-trial-nct07429240","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/precision-biosciences-pbgene-dmd-phase-1-2a-function-dmd-clinical-trial-nct07429240\/","title":{"rendered":"Precision BioSciences, Duchenne Kas Distrofisi Olan Ayakta Tedavi G\u00f6ren Hastalarda PBGENE-DMD'nin Faz 1\/2a (FUNCTION-DMD) \u00c7al\u0131\u015fmas\u0131n\u0131n Ba\u015flad\u0131\u011f\u0131n\u0131 Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Precision BioSciences, 2 ila 7 ya\u015f aras\u0131 ayakta tedavi g\u00f6ren Duchenne kas distrofisi hastalar\u0131nda PBGENE-DMD&#039;nin (NCT07429240) Faz 1\/2a denemesinin ba\u015flat\u0131ld\u0131\u011f\u0131n\u0131 duyurdu.<\/strong> Bu \u00e7al\u0131\u015fmada, FUNCTION-DMD&#039;nin tek dozu 130 hafta boyunca incelenecektir. <strong>Devam\u0131n\u0131 oku:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/precision-biosciences-pbgene-dmd-function-dmd-phase-1-2-clinical-study\/\" target=\"_blank\" rel=\"noreferrer noopener\">Precision BioSciences, ABD&#039;de Ara\u015ft\u0131rma Ama\u00e7l\u0131 Yeni \u0130la\u00e7 Ba\u015fvurusu i\u00e7in FDA Onay\u0131 Ald\u0131<\/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=\"#what-is-pbgene-dmd-phase-1-2-a-clinical-trial\">PBGENE-DMD Faz 1\/2a Klinik \u00c7al\u0131\u015fmas\u0131 nedir?<\/a><\/li><li><a href=\"#pbgene-dmd-phase-1-2-a-nct-07429240\">PBGENE-DMD A\u015fama 1\/2a \u2013 NCT07429240<\/a><\/li><li><a href=\"#how-to-participate-in-the-pbgene-dmd-phase-1-2-a-clinical-trial\">PBGENE-DMD Faz 1\/2a Klinik \u00c7al\u0131\u015fmas\u0131na Nas\u0131l Kat\u0131labilirsiniz?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu Faz 1\/2a \u00e7al\u0131\u015fmas\u0131, 45-55. ekzonlar\u0131n \u00e7\u0131kar\u0131lmas\u0131na uygun mutasyonlara sahip DMD&#039;li bireylerde PBGENE-DMD&#039;nin g\u00fcvenli\u011fini, tolerans\u0131n\u0131 ve erken etkinlik belirtilerini de\u011ferlendirmek \u00fczere tasarlanm\u0131\u015ft\u0131r.<\/strong> Mevcut tedavi se\u00e7eneklerinin s\u0131n\u0131rl\u0131l\u0131klar\u0131 g\u00f6z \u00f6n\u00fcne al\u0131nd\u0131\u011f\u0131nda, PBGENE-DMD, DMD&#039;li ki\u015filerin en b\u00fcy\u00fck molek\u00fcler alt grubunda altta yatan genetik kusurun potansiyel olarak tek seferlik, uzun s\u00fcreli d\u00fczeltilmesini hedefleyen umut vadeden yeni bir strateji sunmaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00e7al\u0131\u015fmaya toplamda en fazla 18 kat\u0131l\u0131mc\u0131 dahil edilebilir. Her kat\u0131l\u0131mc\u0131 i\u00e7in \u00e7al\u0131\u015fma s\u00fcresi toplamda yakla\u015f\u0131k 130 hafta olacakt\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-pbgene-dmd-phase-1-2-a-clinical-trial\">PBGENE-DMD Faz 1\/2a Klinik \u00c7al\u0131\u015fmas\u0131 nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Bu, Duchenne kas distrofisi (DMD) olan ve bu tedaviye yan\u0131t verebilecek spesifik genetik de\u011fi\u015fikliklere sahip erkek \u00e7ocuklarda ve erkeklerde PBGENE-DMD ad\u0131 verilen yeni bir tedaviyi test eden erken a\u015fama bir klinik \u00e7al\u0131\u015fmad\u0131r. Kat\u0131l\u0131mc\u0131lar, tedavinin tek bir dozunu damar yoluyla (koldaki bir damar) alacaklard\u0131r. Doktorlar, g\u00fcvenli\u011fi, yan etkileri ve tedavinin i\u015fe yarad\u0131\u011f\u0131na dair erken belirtileri yak\u0131ndan izleyeceklerdir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Olas\u0131 ba\u011f\u0131\u015f\u0131kl\u0131k reaksiyonlar\u0131n\u0131 azaltmaya yard\u0131mc\u0131 olmak i\u00e7in, kat\u0131l\u0131mc\u0131lara tedavi s\u00fcresi boyunca k\u0131sa s\u00fcreli ila\u00e7lar da verilecektir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fma iki b\u00f6l\u00fcmden olu\u015fmaktad\u0131r. Birinci b\u00f6l\u00fcmde ara\u015ft\u0131rmac\u0131lar g\u00fcvenli ve iyi tolere edilebilen bir doz belirleyeceklerdir. Bu doz onaylan\u0131rsa, ikinci b\u00f6l\u00fcmde tedaviyi daha detayl\u0131 de\u011ferlendirmek i\u00e7in ek kat\u0131l\u0131mc\u0131lar dahil edilecektir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fmaya en fazla 18 kat\u0131l\u0131mc\u0131 dahil edilecek. Her ki\u015fi yakla\u015f\u0131k 2,5 y\u0131l (yakla\u015f\u0131k 130 hafta) boyunca takip edilecek.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <a href=\"https:\/\/dmdwarrior.com\/tr\/faq-pbgene-dmd-gene-editing-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">PBGENE-DMD Gen D\u00fczenleme Terapisi: Faydalar\u0131, Uygunluk Kriterleri ve S\u0131k\u00e7a Sorulan Sorular<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"pbgene-dmd-phase-1-2-a-nct-07429240\">PBGENE-DMD A\u015fama 1\/2a \u2013 NCT07429240<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne Musk\u00fcler Distrofisi Olan Kat\u0131l\u0131mc\u0131larda PBGENE-DMD&#039;nin G\u00fcvenli\u011fini, Tolerans\u0131n\u0131 ve \u00d6n Etkinli\u011fini De\u011ferlendirmek \u0130\u00e7in Faz 1\/2a, \u00c7ok Merkezli, A\u00e7\u0131k Etiketli Bir \u00c7al\u0131\u015fma (FUNCTION-DMD)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fman\u0131n Ba\u015flang\u0131\u00e7 Tarihi (Tahmini):<\/strong> 2026-03<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Birincil Tamamlanma (Tahmini):<\/strong> 2029-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fman\u0131n Tamamlanma Tarihi (Tahmini):<\/strong> 2029-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Tahmini Kat\u0131l\u0131mc\u0131 Say\u0131s\u0131:<\/strong> 18<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fma T\u00fcr\u00fc:<\/strong> Giri\u015fimsel<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Konum:<\/strong> Arkansas \u00c7ocuk Hastanesi<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fmaya Kat\u0131lmaya Uygun Ya\u015f Gruplar\u0131:<\/strong> 2 ila 7 Ya\u015f Aras\u0131 (\u00c7ocuk)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Temel dahil etme ve hari\u00e7 tutma kriterleri. \u2013 <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07429240\" target=\"_blank\" rel=\"noreferrer noopener\">NCT07429240<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-to-participate-in-the-pbgene-dmd-phase-1-2-a-clinical-trial\">PBGENE-DMD Faz 1\/2a Klinik \u00c7al\u0131\u015fmas\u0131na Nas\u0131l Kat\u0131labilirsiniz?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD Faz 1\/2a klinik \u00e7al\u0131\u015fmas\u0131na kat\u0131lmak i\u00e7in a\u015fa\u011f\u0131daki ileti\u015fim bilgilerini kullanabilirsiniz.&nbsp;<strong>Daha fazla bilgi edin<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/how-to-participate-in-clinical-trials-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne (DMD) \u0130\u00e7in Klinik Ara\u015ft\u0131rmalara Nas\u0131l Kat\u0131labilirsiniz?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0130sim:<\/strong> Eileen Bristow<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Telefon Numaras\u0131:<\/strong> +1-610-462-1004<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>E-posta:<\/strong> function-DMD@precisionbiosciences.com<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne \u0130\u00e7in Klinik Ara\u015ft\u0131rmalar (T\u00fcm Ara\u015ft\u0131rmalar\u0131n Listesi)<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences announced the commencement of a Phase 1\/\u200b2a trial of PBGENE-DMD (NCT07429240) in ambulatory patients with Duchenne aged 2 to 7 years. In this trial, single dose of FUNCTION-DMD will be studied over 130 weeks. Read More: Precision BioSciences Receives U.S. FDA Clearance of Investigational New Drug Application This Phase 1\/2a study is designed [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6291,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[137,242,359,479,72,493,314,313],"class_list":["post-6289","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-clinical-trial","tag-clinical-trials","tag-full-length-dystrophin","tag-function-dmd","tag-gene-editing","tag-nct07429240","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6289","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=6289"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6289\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/6291"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=6289"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=6289"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=6289"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}