{"id":6739,"date":"2026-03-24T15:16:30","date_gmt":"2026-03-24T12:16:30","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6739"},"modified":"2026-03-24T15:18:59","modified_gmt":"2026-03-24T12:18:59","slug":"inspire-duchenne-sgt-003-clinical-trial-update","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/inspire-duchenne-sgt-003-clinical-trial-update\/","title":{"rendered":"SGT-003 Gen Terapisi: INSPIRE DUCHENNE Faz 3 \u00c7al\u0131\u015fmas\u0131 Ba\u015flad\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Solid Biosciences, Duchenne kas distrofisi (DMD) i\u00e7in deneysel gen terapisi SGT-003&#039;\u00fc de\u011ferlendiren devam eden Faz 1\/2 INSPIRE DUCHENNE klinik \u00e7al\u0131\u015fmas\u0131ndan cesaret verici ara sonu\u00e7lar bildirdi.<\/strong> Veriler, tedavinin kritik kas proteinlerini geri kazand\u0131rmaya, kas sa\u011fl\u0131\u011f\u0131 biyobelirte\u00e7lerini iyile\u015ftirmeye ve tedavi edilen kat\u0131l\u0131mc\u0131larda kalp fonksiyonunu stabilize etmeye yard\u0131mc\u0131 olabilece\u011fini g\u00f6stermektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">G\u00fcncelleme, 2026 Kas Distrofisi Derne\u011fi (MDA) Y\u0131ll\u0131k Toplant\u0131s\u0131&#039;nda sunuldu ve kas biyopsilerinden, kan biyobelirte\u00e7lerinden, kardiyak de\u011ferlendirmelerden ve g\u00fcvenlik izlemesinden elde edilen yeni bulgular\u0131 vurgulad\u0131.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">INSPIRE DUCHENNE Deneme \u00d6zeti<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>INSPIRE DUCHENNE, Duchenne kas distrofisi olan \u00e7ocuklarda SGT-003&#039;\u00fcn g\u00fcvenli\u011fini, tolerans\u0131n\u0131 ve biyolojik aktivitesini de\u011ferlendirmek \u00fczere tasarlanm\u0131\u015f, insanlarda ilk kez uygulanan Faz 1\/2 klinik denemesidir.<\/strong> \u015eimdi Okuyun: <a href=\"https:\/\/dmdwarrior.com\/tr\/faq-solid-biosciences-sgt-003-gene-therapy\/\" target=\"_blank\" data-type=\"link\" data-id=\"https:\/\/dmdwarrior.com\/faq-solid-biosciences-sgt-003-gene-therapy\/\" rel=\"noreferrer noopener\">Solid Biosciences SGT-003 Gen Terapisi Hakk\u0131nda S\u0131k\u00e7a Sorulan Sorular<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fman\u0131n temel \u00f6zellikleri \u015funlard\u0131r:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Tek doz gen terapisi inf\u00fczyonu<\/strong><\/li>\n\n\n\n<li>Doz seviyesi 1E14 vg\/kg<\/li>\n\n\n\n<li><strong>A\u00e7\u0131k etiketli, \u00e7ok merkezli tasar\u0131m<\/strong><\/li>\n\n\n\n<li>\u00c7al\u0131\u015fma, ABD, Kanada, \u0130talya ve Birle\u015fik Krall\u0131k&#039;ta 15 klinik merkezde y\u00fcr\u00fct\u00fclm\u00fc\u015ft\u00fcr.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>11 Mart 2026 tarihi itibariyle toplam 40 kat\u0131l\u0131mc\u0131 SGT-003 terapisini ald\u0131.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">G\u00fc\u00e7l\u00fc Mikrodistrofin \u0130fadesi G\u00f6zlemlendi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Kas biyopsisi sonu\u00e7lar\u0131, tedavi sonras\u0131nda g\u00fc\u00e7l\u00fc bir mikrodistrofin \u00fcretimi oldu\u011funu g\u00f6sterdi.<\/strong> Mikrodistrofin, Duchenne kas distrofisi olan ki\u015filerde kas h\u00fccrelerini stabilize etmeye yard\u0131mc\u0131 olmak \u00fczere tasarlanm\u0131\u015f, distrofin proteininin k\u0131salt\u0131lm\u0131\u015f bir versiyonudur.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ara\u015ft\u0131rmac\u0131lar ayr\u0131ca distrofinle ili\u015fkili protein kompleksinin (DAPC) temel bile\u015fenlerinin, a\u015fa\u011f\u0131dakiler de dahil olmak \u00fczere, yeniden olu\u015ftu\u011funu g\u00f6zlemlediler:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Beta-sarkoglikan<\/strong><\/li>\n\n\n\n<li><strong>N\u00f6ronal nitrik oksit sentaz (nNOS)<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Bu proteinler kas b\u00fct\u00fcnl\u00fc\u011f\u00fcn\u00fcn korunmas\u0131nda kritik bir rol oynar. Duchenne kas distrofisinde, distrofinin yoklu\u011fu bu kompleksi dengesizle\u015ftirerek kas hasar\u0131na ve dejenerasyonuna yol a\u00e7ar.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">SGT-003, mikrodistrofin yap\u0131s\u0131n\u0131n, nNOS&#039;un kas dokusunda do\u011fru \u015fekilde lokalize olmas\u0131na yard\u0131mc\u0131 olan R16\/R17 ba\u011flanma alan\u0131n\u0131 i\u00e7ermesi nedeniyle dikkat \u00e7ekicidir; bu \u00f6zellik, di\u011fer bir\u00e7ok gen terapisi tasar\u0131m\u0131nda bulunmamaktad\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Biyobelirte\u00e7lerdeki Geli\u015fmeler Daha Sa\u011fl\u0131kl\u0131 Kas Liflerine \u0130\u015faret Ediyor<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fmada ayr\u0131ca kas hasar\u0131 ve onar\u0131m\u0131yla ili\u015fkili \u00e7ok say\u0131da biyobelirte\u00e7 de \u00f6l\u00e7\u00fclm\u00fc\u015ft\u00fcr. Sonu\u00e7lar, SGT-003 tedavisinin kas liflerini stabilize etmeye ve devam eden dejenerasyonu azaltmaya yard\u0131mc\u0131 olabilece\u011fini g\u00f6stermektedir. Daha fazla bilgi edinin: <a href=\"https:\/\/dmdwarrior.com\/tr\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" data-type=\"post\" data-id=\"6725\" rel=\"noreferrer noopener\">Biyobelirte\u00e7ler<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>De\u011ferlendirilen \u00f6nemli belirte\u00e7lerden biri embriyonik miyozin a\u011f\u0131r zinciri (eMHC) idi.<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>eMHC normalde fetal geli\u015fim s\u0131ras\u0131nda ifade edilir.<\/li>\n\n\n\n<li>Duchenne kas distrofisinde, kaslar devam eden hasar\u0131 onarmaya \u00e7al\u0131\u015ft\u0131\u011f\u0131nda yeniden ortaya \u00e7\u0131kar.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ara\u015ft\u0131rmac\u0131lar, 90. g\u00fcnde eMHC pozitif liflerde ortalama 44%&#039;lik bir azalma g\u00f6zlemlediler; bu da tedavinin Duchenne kas distrofisinde s\u0131kl\u0131kla g\u00f6r\u00fclen kas dejenerasyonu ve rejenerasyon d\u00f6ng\u00fcs\u00fcn\u00fc k\u0131rmaya yard\u0131mc\u0131 olabilece\u011fini g\u00f6steriyor.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu biyobelirte\u00e7 iyile\u015ftirmeleri \u015funlar\u0131 g\u00f6stermektedir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Kas hasar\u0131n\u0131n azalmas\u0131<\/li>\n\n\n\n<li>Kas lifi stabilitesinin iyile\u015ftirilmesi<\/li>\n\n\n\n<li>Kas k\u00f6k h\u00fccrelerinin korunmas\u0131<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">Kalp Fonksiyonunun Stabilize Oldu\u011funun Belirtileri<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ba\u015flang\u0131\u00e7ta kalp \u00f6l\u00e7\u00fcmleri yaln\u0131zca g\u00fcvenlik izleme amac\u0131yla dahil edilmi\u015f olsa da, ara\u015ft\u0131rmac\u0131lar tedavi sonras\u0131nda kalp fonksiyonlar\u0131nda stabilizasyon ve iyile\u015fme g\u00f6zlemlediler.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Kalp fonksiyonu, kalbin kan\u0131 ne kadar etkili bir \u015fekilde pompalad\u0131\u011f\u0131n\u0131 \u00f6l\u00e7en sol ventrik\u00fcl ejeksiyon fraksiyonu (LVEF) kullan\u0131larak de\u011ferlendirildi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Geli\u015fmeler \u00f6zellikle tedavi \u00f6ncesinde d\u00fc\u015f\u00fck-normal LVEF seviyelerine sahip kat\u0131l\u0131mc\u0131larda belirgindi.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu bulgu \u00f6zellikle \u00f6nemlidir \u00e7\u00fcnk\u00fc kardiyomiyopati, Duchenne kas distrofisinde \u00f6nde gelen \u00f6l\u00fcm nedenlerinden biridir. \u00c7al\u0131\u015fmalar \u015funu g\u00f6stermektedir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Hastalar\u0131n yakla\u015f\u0131k y\u00fczde 25'inde 6 ya\u015f\u0131na kadar kardiyomiyopati belirtileri g\u00f6r\u00fclmektedir.<\/strong><\/li>\n\n\n\n<li>Yakla\u015f\u0131k y\u00fczde 59'unda 10 ya\u015f\u0131na kadar belirtiler g\u00f6r\u00fclmeye ba\u015flanm\u0131\u015ft\u0131r.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">G\u00fcvenlik ve Tolerans<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>SGT-003 bug\u00fcne kadar olumlu bir g\u00fcvenlik profili sergiledi.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Tedavi edilen 40 kat\u0131l\u0131mc\u0131 aras\u0131nda \u015funlar yer almaktad\u0131r:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Tedavi genel olarak iyi tolere edildi.<\/strong><\/li>\n\n\n\n<li>Ara analizde \u00f6nemli bir g\u00fcvenlik endi\u015fesi bildirilmemi\u015ftir.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Tedavinin bir di\u011fer ay\u0131rt edici \u00f6zelli\u011fi ise basitle\u015ftirilmi\u015f ba\u011f\u0131\u015f\u0131kl\u0131k y\u00f6netimi yakla\u015f\u0131m\u0131d\u0131r. Terapi, sadece steroid i\u00e7eren profilaktik imm\u00fcnomod\u00fclasyon rejimi kullan\u0131larak uygulan\u0131r; bu da di\u011fer baz\u0131 gen terapilerinde kullan\u0131lan daha karma\u015f\u0131k protokollere k\u0131yasla tedavi y\u00fck\u00fcn\u00fc azaltabilir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">SGT-003 Gen Terapisi: INSPIRE DUCHENNE Faz 3 (NCT06138639)<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00e7al\u0131\u015fma, Duchenne kas distrofisi (DMD) hastalar\u0131 i\u00e7in SGT-003 ad\u0131 verilen bir gen terapisini ara\u015ft\u0131rmaktad\u0131r. \u00c7al\u0131\u015fmaya kat\u0131lmak i\u00e7in hastalar\u0131n kar\u015f\u0131lamas\u0131 gereken belirli kriterler bulunmaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <a href=\"https:\/\/dmdwarrior.com\/tr\/how-to-participate-in-clinical-trials-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u00c7ocu\u011fum Duchenne Klinik Denemelerine Nas\u0131l Kat\u0131labilir?<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Kimler Kat\u0131labilir? (Kat\u0131l\u0131m Kriterleri)<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ya\u015f gruplar\u0131:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Grup 1: 4 ila 7 ya\u015f aras\u0131<\/li>\n\n\n\n<li>Grup 2: 7 ila 12 ya\u015f aras\u0131<\/li>\n\n\n\n<li>Grup 3: Do\u011fumdan 4 ya\u015f alt\u0131<\/li>\n\n\n\n<li>Grup 4: 12 ila 18 ya\u015f aras\u0131<\/li>\n\n\n\n<li>Grup 5: 10 ila 18 ya\u015f aras\u0131<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Y\u00fcr\u00fcme yetene\u011fi:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>1., 2. ve 4. Gruplar: Y\u00fcr\u00fcyebilme \u015fart\u0131 aranmaktad\u0131r.<\/li>\n\n\n\n<li>Grup 3: Y\u00fcr\u00fcyebilen veya y\u00fcr\u00fcyemeyen olabilir.<\/li>\n\n\n\n<li>Grup 5: \u015eu anda y\u00fcr\u00fcyemiyor olmal\u0131, ancak ge\u00e7mi\u015fte y\u00fcr\u00fcyebilmi\u015f olmal\u0131d\u0131r.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Ayr\u0131ca, kat\u0131l\u0131mc\u0131lar\u0131n (uygulanabilir durumlarda) 10 metrelik y\u00fcr\u00fcy\u00fc\u015f\u00fc\/ko\u015fuyu 30 saniyenin alt\u0131nda tamamlayabilmeleri gerekmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Te\u015fhis ve genetik:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Duchenne kas distrofisi tan\u0131s\u0131n\u0131n do\u011frulanm\u0131\u015f olmas\u0131 gerekmektedir.<\/li>\n\n\n\n<li>DMD ile uyumlu bir distrofin geni mutasyonuna sahip olmal\u0131d\u0131r.<\/li>\n\n\n\n<li>Baz\u0131 durumlarda, distrofin seviyelerini \u00f6l\u00e7mek i\u00e7in kas biyopsisi gerekebilir.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ba\u011f\u0131\u015f\u0131kl\u0131k durumu:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>AAV (viral vekt\u00f6r) antikorlar\u0131 i\u00e7in negatif test sonucu gereklidir.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Steroid kullan\u0131m\u0131:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>1, 2, 4 ve 5. Gruplar:\n<ul class=\"wp-block-list\">\n<li>En az 12 hafta boyunca d\u00fczenli g\u00fcnl\u00fck steroid tedavisi uygulanmal\u0131d\u0131r.<\/li>\n\n\n\n<li>Bu, prednizonu (en az 0,5 mg\/kg\/g\u00fcn) veya deflazakort&#039;u (en az 0,75 mg\/kg\/g\u00fcn) i\u00e7erir.<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>Grup 3: Steroid gereksinimi yok<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Fiziksel de\u011ferlendirmeler:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>10 metrelik y\u00fcr\u00fcme\/ko\u015fma testi kriterlerini kar\u015f\u0131lamal\u0131d\u0131r.<\/li>\n\n\n\n<li>Yatar pozisyondan aya\u011fa kalkma s\u00fcresi kriterini kar\u015f\u0131lamal\u0131d\u0131r.<\/li>\n\n\n\n<li>Grup 5&#039;in ayr\u0131ca \u00fcst ekstremite fonksiyon gereksinimlerini de kar\u015f\u0131lamas\u0131 gerekir (PUL 2.0 testi).<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Di\u011fer gereksinim:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>V\u00fccut a\u011f\u0131rl\u0131\u011f\u0131 90 kg veya daha az olmal\u0131d\u0131r.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT06138639\" target=\"_blank\" rel=\"noreferrer noopener\">NCT06138639<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h3 class=\"wp-block-heading\">Kimler Kat\u0131lamaz? (Kat\u0131l\u0131m D\u0131\u015f\u0131 B\u0131rakma Kriterleri)<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">A\u015fa\u011f\u0131daki durumlarda kat\u0131l\u0131mc\u0131lar \u00e7al\u0131\u015fmaya kat\u0131lamazlar:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Son 3 ay i\u00e7inde distrofin modifiye edici ila\u00e7lar kulland\u0131m.<\/li>\n\n\n\n<li>Daha \u00f6nce herhangi bir gen terapisi (onayl\u0131 veya deneysel) alm\u0131\u015f olmak.<\/li>\n\n\n\n<li>Son 3 ay i\u00e7inde veya ila\u00e7 v\u00fccuttan tamamen at\u0131lmadan \u00f6nce belirli ila\u00e7lar kullanm\u0131\u015f olmak.<\/li>\n\n\n\n<li>Genetik raporla belgelenmi\u015f ve Sponsor genetik testiyle do\u011frulanm\u0131\u015f, DMD geninde 1 ila 11. eksonlar\u0131 veya 42 ila 45. eksonlar\u0131 veya 57 ila 69. eksonlar\u0131 (dahil) ifade etmeyece\u011fi \u00f6ng\u00f6r\u00fclen herhangi bir delesyon mutasyonu, de\u011fi\u015fmez veya varyant ile ili\u015fkili, yerle\u015fik klinik DMD tan\u0131s\u0131.<\/li>\n<\/ul>\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>Solid Biosciences has reported encouraging interim results from its ongoing Phase 1\/2 INSPIRE DUCHENNE clinical trial, evaluating the investigational gene therapy SGT-003 for Duchenne muscular dystrophy (DMD). The data suggest the therapy may help restore critical muscle proteins, improve biomarkers of muscle health, and stabilize heart function in treated participants. The update was presented at [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6740,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[61,419,524,110,170,109],"class_list":["post-6739","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-gene-therapy","tag-impact-duchenne","tag-inspire-duchenne","tag-sgt-003","tag-sgt-003-gene-therapy","tag-solid-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6739","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=6739"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6739\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/6740"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=6739"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=6739"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=6739"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}