{"id":5673,"date":"2026-02-05T04:47:49","date_gmt":"2026-02-05T01:47:49","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5673"},"modified":"2026-02-07T09:41:46","modified_gmt":"2026-02-07T06:41:46","slug":"elevidys-gene-therapy-efficacy-in-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/elevidys-gene-therapy-efficacy-in-dmd\/","title":{"rendered":"Milyon Dolarl\u0131k Fiyat\u0131na G\u00f6re, Elevidys Gen Terapisi DMD'li \u00c7ocuklar\u0131n Durumunu Ne Kadar \u0130yile\u015ftiriyor?"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Duchenne kas distrofisi (DMD) i\u00e7in FDA taraf\u0131ndan onaylanan tek gen terapisi olan Elevidys, hastalardan elde edilen \u00fc\u00e7 y\u0131ll\u0131k verilerin yay\u0131nlanmas\u0131n\u0131n ard\u0131ndan DMD topluluklar\u0131n\u0131 ve yat\u0131r\u0131mc\u0131lar\u0131 hayal k\u0131r\u0131kl\u0131\u011f\u0131na u\u011fratt\u0131. 2,9 milyon Amerikan dolar\u0131 fiyatla pazarlanan \u00fcr\u00fcn, DMD&#039;li \u00e7ocuklar\u0131n aileleri i\u00e7in \u00e7e\u015fitli sorular\u0131 g\u00fcndeme getirdi: &#039;Elevidys gen terapisi ne kadar etkili, \u00e7ocuklar\u0131n ya\u015fam s\u00fcresini ne kadar uzat\u0131yor ve bir terapinin etkinli\u011fini yaln\u0131zca NSAA skorlar\u0131na dayanarak de\u011ferlendirmek do\u011fru mu?&#039; &gt;&gt;&gt; <strong>Daha fazla bilgi edin:<\/strong> <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 nedir?<\/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=\"#sarepta-really-needs-something-to-freshen-up-the-story\">Sarepta&#039;n\u0131n Hikayeyi Canland\u0131rmak \u0130\u00e7in Ger\u00e7ekten Bir \u015eeye \u0130htiyac\u0131 Var<\/a><\/li><li><a href=\"#i-dont-know-if-elevidys-works\">Elevidys i\u015fe yar\u0131yor mu yaram\u0131yor mu?<\/a><\/li><li><a href=\"#questions-from-families-organizing-the-campaign\">Ba\u011f\u0131\u015f Toplama Etkinlikleri D\u00fczenleyen Ailelerden Gelen Sorular<\/a><\/li><li><a href=\"#which-dmd-gene-therapy-should-you-choose\">Hangi DMD Gen Terapisini Se\u00e7melisiniz?<\/a><\/li><li><a href=\"#sareptas-1-year-share-price\">Sarepta&#039;n\u0131n 1 Y\u0131ll\u0131k Hisse Fiyat\u0131<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"sarepta-really-needs-something-to-freshen-up-the-story\">Sarepta&#039;n\u0131n Hikayeyi Canland\u0131rmak \u0130\u00e7in Ger\u00e7ekten Bir \u015eeye \u0130htiyac\u0131 Var<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Sarepta yak\u0131n zamanda Duchenne kas distrofisi i\u00e7in en \u00f6nemli biyobelirte\u00e7lerden biri olan kreatin kinaz seviyelerini i\u00e7ermeyen veriler yay\u0131nlad\u0131. Elevidys gen terapisine eri\u015fim i\u00e7in kampanya y\u00fcr\u00fcten aileler, bu teknik bilginin payla\u015f\u0131lmamas\u0131na k\u0131zg\u0131n. Terapiye eri\u015fim i\u00e7in yorulmadan fon toplayan ailelerin b\u00fcy\u00fck \u00e7o\u011funlu\u011fu, \u201cSize milyonlarca dolar veren biziz, ama siz bize kar\u015f\u0131l\u0131\u011f\u0131nda hi\u00e7bir \u015fey vermiyorsunuz; ger\u00e7ekleri, verileri ve bilimi. Sadece param\u0131z\u0131 al\u0131yorsunuz.\u201d diyor. <strong>Daha fazla bilgi edin:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/sarepta-3-year-topline-data-of-elevidys-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Sarepta, ELEVIDYS&#039;in 3 Y\u0131ll\u0131k \u00d6zet Verilerini A\u00e7\u0131klad\u0131<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">2025 bahar\u0131nda Elevidys ile ba\u011flant\u0131l\u0131 iki \u00f6l\u00fcm vakas\u0131 ya\u015fan\u0131nca, Sarepta&#039;n\u0131n hisse senedi d\u00fc\u015fmeye ba\u015flad\u0131. Yaz aylar\u0131nda, Elevidys ile ayn\u0131 AAV vekt\u00f6r\u00fcn\u00fc kullanan, uzuv ku\u015fa\u011f\u0131 kas distrofisi i\u00e7in deneysel bir gen tedavisiyle ba\u011flant\u0131l\u0131 \u00fc\u00e7\u00fcnc\u00fc bir \u00f6l\u00fcm vakas\u0131 haberi ortaya \u00e7\u0131kt\u0131. Genel olarak, \u015firketin hisse senedi fiyat\u0131 y\u0131l ba\u015f\u0131ndan itibaren 80% d\u00fc\u015ferek neredeyse on y\u0131l\u0131n en d\u00fc\u015f\u00fck seviyesine geriledi.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"i-dont-know-if-elevidys-works\">Elevidys i\u015fe yar\u0131yor mu yaram\u0131yor mu?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Yeni a\u00e7\u0131klanan 2025 mali tablosu ve Elevidys&#039;ye ili\u015fkin g\u00fcncellenmi\u015f \u00fc\u00e7 y\u0131ll\u0131k istatistiklerin durumu hafifletece\u011fini d\u00fc\u015f\u00fcnen \u015firket y\u00f6netimi hayal k\u0131r\u0131kl\u0131\u011f\u0131na u\u011frayabilir. Sarepta, NSAA&#039;da 4 puanl\u0131k bir art\u0131\u015f\u0131 gen tedavisinin faydal\u0131 oldu\u011funa dair bir g\u00f6sterge olarak g\u00f6rse de, tedavinin maliyeti ve 4 puanl\u0131k NSAA skoru endi\u015feleri art\u0131r\u0131yor. <strong>DMD hastas\u0131 \u00e7ocuklar\u0131n aileleri, Sarepta&#039;dan bu sorulara yan\u0131t bekliyor:<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha Fazlas\u0131n\u0131 Ke\u015ffedin: <a href=\"https:\/\/dmdwarrior.com\/tr\/what-is-creatine-kinase-ck\/\" target=\"_blank\" rel=\"noreferrer noopener\">Kreatin Kinaz (CK) Nedir?<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"questions-from-families-organizing-the-campaign\">Ba\u011f\u0131\u015f Toplama Etkinlikleri D\u00fczenleyen Ailelerden Gelen Sorular<\/h2>\n\n\n\n<ol class=\"wp-block-list\">\n<li>NSAA&#039;daki 4 puanl\u0131k art\u0131\u015f ger\u00e7ekten $2.9 milyon dolara m\u0131 mal oluyor?<\/li>\n\n\n\n<li>\u00dc\u00e7 y\u0131l \u00f6nce, Elevidys tedavisi i\u00e7in aday olan bir DMD&#039;li \u00e7ocu\u011fun kreatin kinaz d\u00fczeyi neydi ve Elevidys tedavisini ald\u0131ktan 3 y\u0131l sonra kreatin kinaz d\u00fczeyi neydi?<\/li>\n\n\n\n<li>Elevidys ald\u0131ktan sonra kreatin kinaz seviyelerinde d\u00fc\u015f\u00fc\u015f olmazsa, bu kas hasar\u0131n\u0131n devam etti\u011fi anlam\u0131na gelmez mi?<\/li>\n\n\n\n<li>Elevidys gen terapisi alan \u00e7ocuklarda kreatin kinaz d\u00fczeylerini \u00f6l\u00e7mek i\u00e7in kas biyopsisi \u00f6rnekleri ald\u0131n\u0131z m\u0131?<\/li>\n<\/ol>\n\n\n\n<p class=\"wp-block-paragraph\">FDA taraf\u0131ndan onaylanan (ancak EMA taraf\u0131ndan onaylanmayan) Duchenne kas distrofisi i\u00e7in ilk gen terapisi olan Elevidys, \u00e7ok yak\u0131nda yeni rakiplerle kar\u015f\u0131 kar\u015f\u0131ya kalacak. Pazara girmeye en yak\u0131n olan \u015firket, ge\u00e7en ay RGX-202 gen terapisi i\u00e7in 2026 y\u0131l\u0131n\u0131n ortalar\u0131na kadar onay ba\u015fvurusunda bulunmay\u0131 planlad\u0131\u011f\u0131n\u0131 a\u00e7\u0131klayan REGENXBIO&#039;dur. SolidBio&#039;nun SGT-003 gen terapisi de iddial\u0131 bir alternatiftir ve bu se\u00e7enekler pazara girerse, DMD hastalar\u0131n\u0131n ve ailelerinin tedavi i\u00e7in Elevidys&#039;den vazge\u00e7meleri \u015fa\u015f\u0131rt\u0131c\u0131 olmaz. &gt;&gt;&gt; <strong>Devam\u0131n\u0131 oku:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/creatine-kinase-ck-dystrophin-level-nsaa-score-in-elevidys-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Elevidys Gen Terapisinde Kreatin Kinaz (CK), Distrofin Seviyesi ve NSAA Skoru<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Sat\u0131\u015flar\u0131n d\u00fc\u015fmesi, rekabetin sertle\u015fmesi ve yeterli net ve g\u00fcven verici bilimsel verinin olmamas\u0131 nedeniyle Sarepta&#039;n\u0131n \u00f6n\u00fcm\u00fczdeki d\u00f6nemde daha da zor zamanlar ge\u00e7irmesi muhtemel. DMDWarrior&#039;\u0131n Alman temsilcisi, biyoteknoloji \u015firketinin markas\u0131n\u0131n zedelenmesi ve hastalar\u0131n\u0131n mutsuz olmas\u0131 nedeniyle, b\u00fcy\u00fck bir ila\u00e7 \u015firketi i\u00e7in bile potansiyel bir sat\u0131n alma hedefi olarak giderek daha az \u00e7ekici hale geldi\u011fini d\u00fc\u015f\u00fcn\u00fcyor. <strong>Kendinize \u015fu sorular\u0131 sorun:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/one-time-gene-therapies-or-exon-skipping-therapies-which-may-be-better-for-duchenne-muscular-dystrophy-dmd-today\/\">\u2018'Tek Seferlik' Gen Terapileri mi yoksa Ekzon Atlama Terapileri mi daha iyi?<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"which-dmd-gene-therapy-should-you-choose\">Hangi DMD Gen Terapisini Se\u00e7melisiniz?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DMD hastalar\u0131 ve aileleri yak\u0131nda zorlu se\u00e7imler yapmak zorunda kalabilir \u00e7\u00fcnk\u00fc Elevidys, REGENXBIO&#039;nun RGX-202 ve Solid Biosciences&#039;nin SGT-003 gibi daha yeni tedavileriyle ayn\u0131 vekt\u00f6r\u00fc kullan\u0131yor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong><em>Kampanya d\u00fczenleyen ailelere tekrar hat\u0131rlat\u0131lmas\u0131 gereken \u015fey \u015fu: \u201cAAV&#039;yi sadece bir kez kullanabilirsiniz!\u201d \u201cDolay\u0131s\u0131yla, Elevidys gen terapisi se\u00e7erseniz, di\u011fer se\u00e7enekleri ka\u00e7\u0131rabilirsiniz.\u201d Bu nedenle, h\u00e2l\u00e2 zaman\u0131 olan ailelerin kampanyalar\u0131n\u0131 yava\u015flatmalar\u0131 veya bir s\u00fcreli\u011fine ertelemeleri iyi bir fikir olabilir.<\/em><\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha Fazlas\u0131n\u0131 Ke\u015ffedin:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Elevidys Hakk\u0131nda S\u0131k\u00e7a Sorulan Sorular<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"sareptas-1-year-share-price\">Sarepta&#039;n\u0131n 1 Y\u0131ll\u0131k Hisse Fiyat\u0131<\/h2>\n\n\n\n<div><a href=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/02\/Sarepta-1-Year-Share-Price-1024x534.jpg\" class=\"td-modal-image\"><figure class=\"wp-block-image size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"1024\" height=\"534\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/02\/Sarepta-1-Year-Share-Price-1024x534.jpg\" alt=\"Sarepta Therapeutics 1 Y\u0131ll\u0131k Hisse Fiyat\u0131\" class=\"wp-image-6177\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/02\/Sarepta-1-Year-Share-Price-1024x534.jpg 1024w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/02\/Sarepta-1-Year-Share-Price-300x157.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/02\/Sarepta-1-Year-Share-Price-768x401.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/02\/Sarepta-1-Year-Share-Price-18x9.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/02\/Sarepta-1-Year-Share-Price-805x420.jpg 805w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/02\/Sarepta-1-Year-Share-Price-150x78.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/02\/Sarepta-1-Year-Share-Price-696x363.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/02\/Sarepta-1-Year-Share-Price-1068x557.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/02\/Sarepta-1-Year-Share-Price.jpg 1290w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" \/><\/figure><\/a><\/div>\n\n\n\n<p class=\"wp-block-paragraph\">EMBARK g\u00fcncellemesinin cesaret verici olmas\u0131na ra\u011fmen, Sarepta Therapeutics&#039;nin son d\u00f6nemdeki hisse senedi performans\u0131 zay\u0131f kald\u0131; 7 g\u00fcnl\u00fck hisse senedi getirisi 8,53% ve y\u0131l ba\u015f\u0131ndan bu yana hisse senedi getirisinde 4,83%&#039;lik bir d\u00fc\u015f\u00fc\u015f ya\u015fand\u0131. Bu arada, 1 y\u0131ll\u0131k toplam hissedar getirisi d\u00fc\u015f\u00fc\u015f\u00fc 82,22% ve 3 y\u0131ll\u0131k toplam hissedar getirisi d\u00fc\u015f\u00fc\u015f\u00fc 83,21%, 90 g\u00fcnl\u00fck hisse senedi getirisinin 8,62% olmas\u0131 k\u0131sa vadeli bir ivme kazanmaya \u00e7al\u0131\u015f\u0131ld\u0131\u011f\u0131n\u0131 g\u00f6sterse bile, piyasa duyarl\u0131l\u0131\u011f\u0131n\u0131n ne kadar so\u011fudu\u011funu vurguluyor.<\/p>","protected":false},"excerpt":{"rendered":"<p>Elevidys, the only gene therapy approved by the FDA for Duchenne muscular dystrophy (DMD), has disappointed DMD communities and investors following the release of three years of data from patients. Marketed at a price of $2.9 million American dollars, the product has raised several questions for families of children with DMD: &#8216;How effective is Elevidys [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6174,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[33,179,61,107,108,65,110,109],"class_list":["post-5673","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-elevidys","tag-elevidys-gene-therapy","tag-gene-therapy","tag-regenxbio","tag-rgx-202","tag-sarepta","tag-sgt-003","tag-solid-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5673","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=5673"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5673\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/6174"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5673"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5673"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5673"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}