{"id":2617,"date":"2025-12-27T21:03:09","date_gmt":"2025-12-27T18:03:09","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=2617"},"modified":"2025-12-27T21:38:43","modified_gmt":"2025-12-27T18:38:43","slug":"duchenne-muscular-dystrophy-researches-to-watch-in-2026","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/duchenne-muscular-dystrophy-researches-to-watch-in-2026\/","title":{"rendered":"2026&#039;da Takip Etmeniz Gereken Alt\u0131 DMD Ara\u015ft\u0131rmas\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Son on y\u0131lda DMD hastalar\u0131 i\u00e7in \u00f6nemli ilerlemeler kaydedilmi\u015f olmas\u0131na ra\u011fmen, h\u00e2l\u00e2 kar\u015f\u0131lanmam\u0131\u015f \u00f6nemli bir talep bulunmaktad\u0131r. Bu ihtiyac\u0131 kar\u015f\u0131lamak amac\u0131yla, Satellos, Regenxbio, Wave, Dyne ve Avidity gibi bir dizi \u015firket, etkinli\u011fi art\u0131rmay\u0131 ve daha geni\u015f eri\u015filebilirli\u011fi hedefleyen deneysel tedaviler geli\u015ftirmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">2026, Duchenne kas distrofisi alan\u0131nda bir at\u0131l\u0131m y\u0131l\u0131 olabilir. Son y\u0131llarda piyasaya bir\u00e7ok tedavi y\u00f6ntemi girmi\u015f olsa da, DMD ile ya\u015fayan \u00e7ocuklar i\u00e7in hala \u00f6nemli bir kar\u015f\u0131lanmam\u0131\u015f ihtiya\u00e7 bulunmaktad\u0131r. Ancak bir\u00e7ok \u015firketin yeni nesil tedaviler geli\u015ftirmesi ve sekt\u00f6r\u00fcn art\u0131k d\u00fczenleyici yollar\u0131 olu\u015fturmas\u0131yla birlikte, uzmanlar yeni se\u00e7eneklerin ufukta oldu\u011funu s\u00f6yl\u00fcyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Satellos, Dyne, Avidity ve Wave Life Sciences gibi bir\u00e7ok \u015firket \u015fu anda bu ihtiyaca yan\u0131t vermek i\u00e7in \u00e7al\u0131\u015f\u0131yor. Yeni nesil ekson atlama terapilerinin ve Regenxbio ile Solid Bio taraf\u0131ndan sunulan gen terapilerinin geli\u015ftirilmesi g\u00f6z \u00f6n\u00fcne al\u0131nd\u0131\u011f\u0131nda, yak\u0131nda daha fazla DMD tedavisinin mevcut olabilece\u011fini d\u00fc\u015f\u00fcnmek i\u00e7in iyi bir neden var. &gt;&gt; Daha Fazlas\u0131n\u0131 Okuyun: <a href=\"https:\/\/dmdwarrior.com\/tr\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD \u0130\u00e7in Yeni Nesil Ekson Atlama Terapileri<\/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=\"#satellos-sat-3247\">Satellos: SAT-3247<\/a><\/li><li><a href=\"#regenxbios-rgx-202\">Regenxbio: RGX-202<\/a><\/li><li><a href=\"#avidity-biosciences-del-zota\">Avidity Biosciences: Del-Zota<\/a><\/li><li><a href=\"#capricor-therapeutics-deramiocel\">Capricor Therapeutics: Deramiocel<\/a><\/li><li><a href=\"#dyne-therapeutics-dyne-251\">Dyne Therapeutics: Dyne-251<\/a><\/li><li><a href=\"#wave-life-sciences-wve-n-531\">Wave Life Sciences: WVE-N531<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"satellos-sat-3247\">Satellos: SAT-3247<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Satellos, 7 ila 10 ya\u015f aras\u0131 ayakta tedavi g\u00f6ren Duchenne kas distrofisi hastalar\u0131nda SAT-3247&#039;nin (NCT07287189) Faz 2A \u00e7al\u0131\u015fmas\u0131n\u0131n ba\u015flat\u0131ld\u0131\u011f\u0131n\u0131 duyurdu. Bu \u00e7al\u0131\u015fmada, SAT-3247&#039;nin iki dozu, optimal dozu, g\u00fcvenli\u011fi, tolerans\u0131 ve \u00f6n etkinli\u011fi belirlemek amac\u0131yla, randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc, haftal\u0131k uygulama rejimiyle 12 hafta boyunca incelenecektir. &gt;&gt; <strong>Daha fazla bilgi edin:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/satellos-sat-3247-phase-2-basecamp-clinical-trial-nct07287189\/\" target=\"_blank\" rel=\"noreferrer noopener\">SAT-3247&#039;nin Faz 2A (BASECAMP) \u00c7al\u0131\u015fmas\u0131<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"regenxbios-rgx-202\">Regenxbio: RGX-202<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eu anda kritik bir ara\u015ft\u0131rma a\u015famas\u0131nda olan RGX-202 ile Regenxbio, gen terapisi pazar\u0131nda Sarepta&#039;n\u0131n Elevidys&#039;si ile rekabet ediyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Deney i\u00e7in bir ya\u015f ve \u00fczeri otuz hasta al\u0131n\u0131yor ve bu da toplam pop\u00fclasyonu Elevidys&#039;nin mevcut etiketinden daha gen\u00e7 hale getiriyor. Kelly&#039;ye g\u00f6re, Kas\u0131m 2024&#039;te yay\u0131nlanan k\u00fc\u00e7\u00fck bir hasta grubundan elde edilen ilk bulgular &quot;son derece cesaret verici&quot;ydi ve sa\u011flam bir g\u00fcvenlik profiliyle birlikte g\u00fc\u00e7 ve zaman fonksiyon testlerinde iyile\u015fmeler g\u00f6steriyordu. Regenxbio, 2026&#039;da bir BLA sunmay\u0131 \u00f6ng\u00f6r\u00fcyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Kelly&#039;ye g\u00f6re, AAV iletim arac\u0131 i\u00e7in \u00e7ok b\u00fcy\u00fck olan DMD geninin boyutu, bu alanda gen terapisi i\u00e7in b\u00fcy\u00fck bir engeldir. Sonu\u00e7 olarak, k\u0131salt\u0131lm\u0131\u015f bir form kullan\u0131l\u0131r, ancak son \u00fcr\u00fcn\u00fc tam boyutlu protein kadar iyi i\u015flev g\u00f6remez. Elevidys ile kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda, Regenxbio&#039;nun mimarisi genin biraz daha b\u00fcy\u00fck bir versiyonuna izin verir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Kelly, daha \u00f6nce yap\u0131lan bir denemenin sonu\u00e7lar\u0131n\u0131n daha fazla say\u0131da hastaya yay\u0131lmas\u0131n\u0131 heyecanla bekliyor. Etkili olursa, DMD gen terapisi arayan ebeveynlere ba\u015fka bir se\u00e7enek ve \u015fans sunaca\u011f\u0131n\u0131 d\u00fc\u015f\u00fcn\u00fcyor. <strong>Daha fazla bilgi edin:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/regenxbio-announces-completion-pivotal-enrollment-and-initiates\/\" target=\"_blank\" rel=\"noreferrer noopener\">REGENXBIO, RGX-202 Pivotal Kayd\u0131n\u0131n Tamamland\u0131\u011f\u0131n\u0131 ve Duchenne Gen Terapisi Program\u0131nda Ticari \u00dcretime Ba\u015flad\u0131\u011f\u0131n\u0131 Duyurdu<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"avidity-biosciences-del-zota\">Avidity Biosciences: Del-Zota<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Avidity, \u015fu anda onayl\u0131 bir ilac\u0131 bulunmayan ekzon 44&#039;e odaklanarak, ekzon 44 atlamas\u0131na yatk\u0131n olan DMD hastalar\u0131n\u0131n 6%&#039;sine tedavi sa\u011flayabilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">CureDuchenne&#039;in ba\u015f bilim g\u00f6revlisi Michael Kelly&#039;ye g\u00f6re, delpacibart zotadirsen (del-zota), bir Faz I\/II denemesinde DMD hastalar\u0131nda distrofin \u00fcretimini \u015fa\u015f\u0131rt\u0131c\u0131 bir \u015fekilde 25% normal i\u015fleve y\u00fckseltti. Bu, herhangi bir ila\u00e7tan g\u00f6zlemlenen en iyi ekzon atlama kan\u0131tlar\u0131ndan biridir. Ek olarak, Del-Zota kreatin kinaz\u0131n\u0131 neredeyse normal seviyelere d\u00fc\u015f\u00fcrd\u00fc.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket, 2026 y\u0131l\u0131n\u0131n ortalar\u0131nda BLA ba\u015fvurusunda bulunmay\u0131 planl\u0131yor ve Explore44 a\u00e7\u0131k etiketli uzatma \u00e7al\u0131\u015fmas\u0131ndan daha fazla sonucun bu y\u0131l i\u00e7inde elde edilmesi bekleniyor. Del-Zota, FDA&#039;den zaten h\u0131zl\u0131 onay s\u0131n\u0131fland\u0131rmas\u0131 ve yetim ila\u00e7 stat\u00fcs\u00fc alm\u0131\u015ft\u0131. <strong>Okumaya devam edin<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/avidity-biosciences-announces-u-s-managed-access-program-map-for-investigational-therapy-del-zota-in-dmd44\/\" target=\"_blank\" rel=\"noreferrer noopener\">Avidity Biosciences, Ara\u015ft\u0131rma Terapisi del-zota (Ekzon 44 Atlama) i\u00e7in ABD Y\u00f6netilen Eri\u015fim Program\u0131n\u0131 (MAP) Duyurdu<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"capricor-therapeutics-deramiocel\">Capricor Therapeutics: Deramiocel<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">FDA \u015fu anda DMD kardiyomiyopatisi i\u00e7in allojenik h\u00fccre tedavisi olan Capricor&#039;ye y\u00f6nelik biyolojik \u00fcr\u00fcn lisans ba\u015fvurusunu inceliyor. &gt;&gt; <strong>Devam\u0131n\u0131 oku:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/capricor-therapeutics-announces-positive-topline-results\/\" data-type=\"link\" data-id=\"https:\/\/dmdwarrior.com\/capricor-therapeutics-announces-positive-topline-results\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD&#039;de Deramiocel&#039;nin De\u011ferlendirilmesine Y\u00f6nelik Kritik Faz 3 HOPE-3 \u00c7al\u0131\u015fmas\u0131<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Onaylan\u0131rsa, Deramiocel, DMD kardiyomiyopatisini \u00f6zel olarak tedavi eden ilk ila\u00e7 olacakt\u0131r. Hastalar\u0131n neredeyse tamam\u0131 yeti\u015fkinlik d\u00f6nemine kadar kalp sorunlar\u0131 geli\u015ftirir ve bu da hastal\u0131\u011f\u0131n ba\u015fl\u0131ca \u00f6l\u00fcm nedenidir. Kurumun verdi\u011fi bilgilere dayanarak, Capricor, DMD pazar\u0131nda al\u0131\u015f\u0131lmad\u0131k bir durum olan tam FDA onay\u0131 i\u00e7in ba\u015fvuruda bulunmu\u015ftur; \u00e7\u00fcnk\u00fc \u00e7o\u011fu ila\u00e7 FDA&#039;nin h\u0131zland\u0131r\u0131lm\u0131\u015f onay program\u0131 arac\u0131l\u0131\u011f\u0131yla onaylanmaktad\u0131r. <strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/what-is-deramiocel\/\" target=\"_blank\" rel=\"noreferrer noopener\">Deramiocel Nedir?<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"dyne-therapeutics-dyne-251\">Dyne Therapeutics: Dyne-251<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Yeni nesil ekson 51 atlama terapisi olan ve FDA girdisine dayanan Dyne-251 i\u00e7in h\u0131zl\u0131 onay almak amac\u0131yla Dyne, \u015fu anda \u00f6nemli bir klinik \u00e7al\u0131\u015fma i\u00e7in hasta al\u0131m\u0131na ba\u015flad\u0131. Distrofin ekspresyonu, dolayl\u0131 bir son nokta olarak kullan\u0131lacakt\u0131r. <strong>Devam\u0131n\u0131 oku:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/dyne-therapeutics-to-apply-for-accelerated-approval-in-q2-2026-for-exon-51-skipping-therapy-dyne-251-z-rostudirsen\/\" target=\"_blank\" rel=\"noreferrer noopener\">Dyne Therapeutics, Exon 51 Atlama Terapisi (DYNE-251) i\u00e7in 2026'n\u0131n 2. \u00c7eyre\u011finde H\u0131zland\u0131r\u0131lm\u0131\u015f Onay i\u00e7in Ba\u015fvuruda Bulunacak<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"wave-life-sciences-wve-n-531\">Wave Life Sciences: WVE-N531<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Wave Life Sciences CEO&#039;su Paul Bolno, hastalar\u0131n ihtiya\u00e7lar\u0131n\u0131 de\u011ferlendirdikten sonra, daha ba\u015far\u0131l\u0131 bir tedavinin yolunun, daha y\u00fcksek distrofin \u00fcretimi ve tutarl\u0131 ifade i\u00e7in h\u00fccre da\u011f\u0131l\u0131m\u0131n\u0131n iyile\u015ftirilmesinden ge\u00e7ti\u011fini s\u00f6yledi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Faz II \u00e7al\u0131\u015fmas\u0131n\u0131n alt\u0131 ayl\u0131k ara analizi, Wave&#039;in WVE-N531 (ekson 53 atlamas\u0131) varyant\u0131n\u0131n t\u00fcm hastalarda tutarl\u0131 bir \u015fekilde 9% distrofin ekspresyonu g\u00f6sterdi\u011fini ortaya koydu. Bolno&#039;ya g\u00f6re bu durum, daha sa\u011fl\u0131kl\u0131 g\u00f6r\u00fcnen kaslara ve kas hasar\u0131n\u0131n bir biyobelirteci olan kreatin kinaz\u0131n kan seviyelerinde azalmaya yol a\u00e7t\u0131. <strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/wve-n531-exon-skipping-therapy-phase-2-clinical-trial-nct07209332\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Musk\u00fcler Distrofisi i\u00e7in WVE-N531 Ekzon Atlama Terapisi Faz 2 Klinik \u00c7al\u0131\u015fmas\u0131 Ba\u015fl\u0131yor<\/a><\/p>\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>Despite significant advancements over the past ten years for DMD patients, there is still a significant unmet demand. In order to meet the need, a number of businesses, such as Satellos, Regenxbio, Wave, Dyne, and Avidity, are developing experimental treatments that aim for increased effectiveness and wider accessibility. 2026 could be a breakout year for [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5426,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[126,87,66,208,77,61,107,251,252,153],"class_list":["post-2617","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-avidity-biosciences","tag-capricor-therapeutics","tag-duchenne-muscular-dystrophy","tag-dyne-therapeutics","tag-exon-skipping","tag-gene-therapy","tag-regenxbio","tag-sat-3247","tag-satellos","tag-wave-life-sciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/2617","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=2617"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/2617\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5426"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=2617"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=2617"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=2617"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}