{"id":7667,"date":"2026-04-22T19:42:28","date_gmt":"2026-04-22T16:42:28","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=7667"},"modified":"2026-04-22T19:42:30","modified_gmt":"2026-04-22T16:42:30","slug":"ck-levels-after-duchenne-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/ck-levels-after-duchenne-gene-therapy\/","title":{"rendered":"Duchenne Gen Terapisinde CK, AST ve ALT Verilerinin Videolardan Daha \u00d6nemli Olmas\u0131n\u0131n Sebebi"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Duchenne kas distrofisi (DMD) i\u00e7in gen terapisi alan\u0131ndaki geli\u015fen ortamda, bir konu giderek daha belirgin hale geliyor: <strong>Duchenne gen terapisi sonras\u0131 CK d\u00fczeyleri, internette dola\u015fan viral videolardan \u00e7ok daha \u00f6nemlidir.<\/strong>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Aileler, tedavi sonras\u0131nda iyile\u015fmeyi g\u00f6steren, duygusal olarak olduk\u00e7a etkileyici, h\u0131zland\u0131r\u0131lm\u0131\u015f videolara maruz kal\u0131yorlar. <strong>Ancak bu g\u00f6rsel anlat\u0131lar genellikle, \u00f6zellikle etkinlik ve g\u00fcvenli\u011fin de\u011ferlendirilmesi i\u00e7in gerekli olan Kreatin Kinaz (CK), AST ve ALT d\u00fczeyleri gibi biyokimyasal veriler e\u015flik etmeden sunulmaktad\u0131r.<\/strong> <\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu durum kritik bir soruyu g\u00fcndeme getiriyor:<br><strong>Tedavi sonu\u00e7lar\u0131n\u0131 belirlemesi gereken biyobelirte\u00e7 verileri yerine neden videolar izliyoruz?<\/strong><\/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=\"#the-problem-viral-videos-are-replacing-clinical-evidence\">Sorun: Viral Videolar Klinik Kan\u0131tlar\u0131n Yerini Al\u0131yor<\/a><\/li><li><a href=\"#ck-levels-after-duchenne-gene-therapy-the-core-biomarker\">Duchenne Gen Terapisi Sonras\u0131 CK D\u00fczeyleri: Temel Biyobelirte\u00e7<\/a><\/li><li><a href=\"#ast-and-alt-essential-safety-and-muscle-biomarkers\">AST ve ALT: Temel G\u00fcvenlik ve Kas Biyobelirte\u00e7leri<\/a><\/li><li><a href=\"#why-are-ck-levels-after-duchenne-gene-therapy-not-fully-disclosed\">Duchenne Gen Terapisi Sonras\u0131nda CK D\u00fczeyleri Neden Tam Olarak A\u00e7\u0131klanm\u0131yor?<\/a><\/li><li><a href=\"#the-ethical-issue-emotion-vs-evidence\">Etik Sorun: Duygu mu, Kan\u0131t m\u0131?<\/a><\/li><li><a href=\"#what-real-transparency-should-look-like\">Ger\u00e7ek \u015eeffafl\u0131k Nas\u0131l Olmal\u0131?<\/a><\/li><li><a href=\"#the-consequences-of-continued-opacity\">Mant\u0131ks\u0131zl\u0131\u011f\u0131n Devam Etmesinin Sonu\u00e7lar\u0131<\/a><\/li><li><a href=\"#frequently-asked-questions-faq\">S\u0131k\u00e7a Sorulan Sorular (SSS)<\/a><\/li><li><a href=\"#final-thoughts\">Son D\u00fc\u015f\u00fcnceler<\/a><\/li><li><a href=\"#academic-sources-and-references\">Akademik Kaynaklar ve Referanslar<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"the-problem-viral-videos-are-replacing-clinical-evidence\">Sorun: Viral Videolar Klinik Kan\u0131tlar\u0131n Yerini Al\u0131yor<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"the-rise-of-anecdotal-evidence-in-dmd\">DMD&#039;de Anekdot Niteli\u011finde Kan\u0131tlar\u0131n Y\u00fckseli\u015fi<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Gen terapisi sonras\u0131nda \u00e7ocuklar\u0131n y\u00fcr\u00fcd\u00fc\u011f\u00fcn\u00fc, merdiven \u00e7\u0131kt\u0131\u011f\u0131n\u0131 veya desteksiz ayakta durdu\u011funu g\u00f6steren videolar yayg\u0131n olarak payla\u015f\u0131l\u0131yor. Bu anlar ger\u00e7ek olsa da, tedavi ba\u015far\u0131s\u0131n\u0131n bilimsel olarak g\u00fcvenilir g\u00f6stergeleri de\u011fildir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eunlardan yoksunlar:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Kontroll\u00fc ko\u015fullar<\/li>\n\n\n\n<li>Standartla\u015ft\u0131r\u0131lm\u0131\u015f fonksiyonel puanlama<\/li>\n\n\n\n<li>Temel kar\u015f\u0131la\u015ft\u0131rmalar<\/li>\n\n\n\n<li>Uzun s\u00fcreli takip<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>En \u00f6nemlisi, Duchenne kas distrofisi i\u00e7in gen terapisi sonras\u0131nda kas hasar\u0131n\u0131n ger\u00e7ekten azal\u0131p azalmad\u0131\u011f\u0131n\u0131 belirlemek i\u00e7in gerekli olan CK d\u00fczeylerine ve normal distrofin ekspresyonuna ili\u015fkin verilere sahip de\u011filler.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <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) Nedir?<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"why-this-is-dangerous\">Bu Neden Tehlikeli?<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Nesnel verilerin yerini anekdot niteli\u011findeki bilgiler ald\u0131\u011f\u0131nda:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Aileler ger\u00e7ek\u00e7i olmayan beklentiler geli\u015ftirebilirler.<\/li>\n\n\n\n<li>Tedavi kararlar\u0131 eksik bilgilerden etkilenebilir.<\/li>\n\n\n\n<li>Bilimsel titizlik zay\u0131flat\u0131l\u0131yor.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu bir ileti\u015fim sorunu de\u011fil, veri \u015feffafl\u0131\u011f\u0131 sorunudur.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"ck-levels-after-duchenne-gene-therapy-the-core-biomarker\">Duchenne Gen Terapisi Sonras\u0131 CK D\u00fczeyleri: Temel Biyobelirte\u00e7<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"what-ck-levels-represent\">CK Seviyeleri Ne Anlama Geliyor?<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Kreatin Kinaz (CK), kas lifleri hasar g\u00f6rd\u00fc\u011f\u00fcnde sal\u0131nan kritik bir enzimdir. DMD&#039;de:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>CK seviyeleri genellikle son derece y\u00fcksektir.<\/strong><\/li>\n\n\n\n<li>S\u00fcrekli y\u00fckselme, devam eden kas dejenerasyonunu yans\u0131t\u0131r.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Gen terapisi sonras\u0131nda, a\u015fa\u011f\u0131daki hususlar\u0131n belirlenmesi i\u00e7in CK d\u00fczeyleri yak\u0131ndan izlenmelidir:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Kas hasar\u0131 azal\u0131yor mu?<\/li>\n\n\n\n<li>Bu tedavi biyolojik bir etki yarat\u0131yor mu?<\/li>\n\n\n\n<li>Hastal\u0131k s\u00fcreci de\u011fi\u015ftiriliyor mu?<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"why-ck-data-must-be-public\">CK Verilerinin Neden Kamuoyuna A\u00e7\u0131k Olmas\u0131 Gerekiyor?<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne gen terapisi sonras\u0131nda CK d\u00fczeylerine eri\u015fim olmamas\u0131 durumunda:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0130yile\u015fme iddialar\u0131 hen\u00fcz do\u011frulanamaz.<\/li>\n\n\n\n<li>\u0130\u015flevsel kazan\u0131mlar biyolojik de\u011fi\u015fimle ili\u015fkilendirilemez.<\/li>\n\n\n\n<li>Hekimler ve aileler bilin\u00e7li kararlar veremez.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>CK iste\u011fe ba\u011fl\u0131 de\u011fil, temel bir gerekliliktir.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"ast-and-alt-essential-safety-and-muscle-biomarkers\">AST ve ALT: Temel G\u00fcvenlik ve Kas Biyobelirte\u00e7leri<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"beyond-liver-function\">Karaci\u011fer Fonksiyonunun \u00d6tesinde<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">AST (Aspartat Aminotransferaz) ve ALT (Alanin Aminotransferaz) s\u0131kl\u0131kla yaln\u0131zca karaci\u011fer enzimleri olarak yanl\u0131\u015f anla\u015f\u0131lmaktad\u0131r. <strong>DMD&#039;de bunlar ayn\u0131 zamanda kas b\u00fct\u00fcnl\u00fc\u011f\u00fcn\u00fc de yans\u0131t\u0131r.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Gen terapisi sonras\u0131nda:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Y\u00fcksek AST\/ALT de\u011ferleri kas y\u0131k\u0131m\u0131na veya tedaviye ba\u011fl\u0131 toksisiteye i\u015faret edebilir.<\/strong><\/li>\n\n\n\n<li>Bu g\u00f6stergelerdeki de\u011fi\u015fiklikler kritik g\u00fcvenlik sinyalleri sa\u011flar.<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"the-transparency-gap\">\u015eeffafl\u0131k A\u00e7\u0131\u011f\u0131<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u00d6nemlerine ra\u011fmen:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>AST ve ALT verileri tutarl\u0131 bir \u015fekilde a\u00e7\u0131klanmamaktad\u0131r.<\/li>\n\n\n\n<li>Kamuoyuna yap\u0131lan a\u00e7\u0131klamalar genellikle eksik veya gecikmelidir.<\/li>\n\n\n\n<li>Aileler eksiksiz bir g\u00fcvenlik profili olmadan kal\u0131yor.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Y\u00fcksek riskli tedavilerde bu \u015feffafl\u0131k eksikli\u011fi kabul edilemez.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"why-are-ck-levels-after-duchenne-gene-therapy-not-fully-disclosed\">Duchenne Gen Terapisi Sonras\u0131nda CK D\u00fczeyleri Neden Tam Olarak A\u00e7\u0131klanm\u0131yor?<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"the-accountability-problem\">Hesap Verebilirlik Sorunu<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Gen terapileri geli\u015ftiren ila\u00e7 \u015firketleri son derece rekabet\u00e7i bir ortamda faaliyet g\u00f6stermektedir. Ancak bu, hayati \u00f6neme sahip biyobelirte\u00e7 verilerinin gizlenmesini hakl\u0131 \u00e7\u0131karmaz.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirketler \u015fu durumlarda:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>G\u00f6rsel iyile\u015ftirmeleri (fonksiyonel) vurguluyor<\/li>\n\n\n\n<li>Se\u00e7tikleri sonu\u00e7lar\u0131 payla\u015f\u0131yor<\/li>\n\n\n\n<li>Biyobelirte\u00e7lerin a\u00e7\u0131klanmas\u0131n\u0131 geciktiriyor<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu durum, hem hastalar\u0131 hem de hekimleri dezavantajl\u0131 duruma d\u00fc\u015f\u00fcren bilgi asimetrisi yarat\u0131r.<\/strong><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"the-role-of-regulators\">D\u00fczenleyicilerin Rol\u00fc<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Avrupa \u0130la\u00e7 Ajans\u0131 gibi kurulu\u015flar g\u00fcvenlik ve \u015feffafl\u0131\u011f\u0131 sa\u011flamaktan sorumludur. Ancak, kamuoyunun \u015funlara eri\u015fimi k\u0131s\u0131tl\u0131d\u0131r:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>CK seviyeleri<\/li>\n\n\n\n<li>AST\/ALT verileri<\/li>\n\n\n\n<li>Uzun vadeli sonu\u00e7lar<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Bir\u00e7ok durumda s\u0131n\u0131rl\u0131 kalmaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>D\u00fczenleyici denetim, ger\u00e7ek zamanl\u0131 veri \u015feffafl\u0131\u011f\u0131na \u00f6ncelik verecek \u015fekilde geli\u015fmelidir.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"the-ethical-issue-emotion-vs-evidence\">Etik Sorun: Duygu mu, Kan\u0131t m\u0131?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Ailelerin video payla\u015fmas\u0131 as\u0131l sorun de\u011fil; eri\u015filebilir veri eksikli\u011fine bir yan\u0131t niteli\u011finde. Ancak bu videolar anlat\u0131n\u0131n merkezine oturdu\u011funda:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0130stemeden ba\u015fkalar\u0131n\u0131 yan\u0131ltabilirler.<\/li>\n\n\n\n<li>\u00d6l\u00e7\u00fclebilir sonu\u00e7lardan uzakla\u015ft\u0131r\u0131r.<\/li>\n\n\n\n<li>Bu durum hasta toplulu\u011fu i\u00e7inde bask\u0131 yarat\u0131r.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">As\u0131l sorun \u015fu ki, sistemde \u015funlar var:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Veriler yetersiz.<\/li>\n\n\n\n<li>Duygu, bo\u015flu\u011fu dolduruyor.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu dengesizlik d\u00fczeltilmelidir.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-real-transparency-should-look-like\">Ger\u00e7ek \u015eeffafl\u0131k Nas\u0131l Olmal\u0131?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">G\u00fcveni ve bilimsel d\u00fcr\u00fcstl\u00fc\u011f\u00fc yeniden tesis etmek i\u00e7in, alan\u0131n yeni bir standart benimsemesi gerekiyor:<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"1-routine-disclosure-of-ck-levels-after-duchenne-gene-therapy\">1. Duchenne Gen Terapisi Sonras\u0131nda CK D\u00fczeylerinin Rutin Olarak A\u00e7\u0131klanmas\u0131<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Ba\u015flang\u0131\u00e7 durumu ile tedavi sonras\u0131 durum kar\u015f\u0131la\u015ft\u0131rmalar\u0131<\/li>\n\n\n\n<li>Boylamsal takip (aylar\/y\u0131llar)<\/li>\n\n\n\n<li>Trendlerin net yorumlanmas\u0131<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"2-full-reporting-of-ast-and-alt\">2. AST ve ALT&#039;nin Tam Raporlanmas\u0131<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>G\u00fcvenlik izleme<\/li>\n\n\n\n<li>Olumsuz olay tespiti<\/li>\n\n\n\n<li>Ba\u011flamsal a\u00e7\u0131klama<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"3-public-access-to-data\">3. Verilere Kamu Eri\u015fimi<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>A\u00e7\u0131k kay\u0131tlar<\/li>\n\n\n\n<li>\u0130ndirilebilir veri k\u00fcmeleri<\/li>\n\n\n\n<li>G\u00f6rsel kontrol panelleri<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"4-independent-validation\">4. Ba\u011f\u0131ms\u0131z Do\u011frulama<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u00dc\u00e7\u00fcnc\u00fc taraf analizi<\/li>\n\n\n\n<li>Akademik i\u015fbirli\u011fi<\/li>\n\n\n\n<li>D\u00fczenleyici inceleme<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"the-consequences-of-continued-opacity\">Mant\u0131ks\u0131zl\u0131\u011f\u0131n Devam Etmesinin Sonu\u00e7lar\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne gen terapisi sonras\u0131nda CK d\u00fczeyleri a\u00e7\u0131klanmazsa:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Gen terapisine olan g\u00fcven azalacak.<\/li>\n\n\n\n<li>Aileler yanl\u0131\u015f bilgilere dayanarak kararlar verebilirler.<\/li>\n\n\n\n<li>Bilimsel ilerleme yava\u015flayabilir.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u015eeffafl\u0131k sadece bilimsel bir gereklilik de\u011fil, ayn\u0131 zamanda etik bir y\u00fck\u00fcml\u00fcl\u00fckt\u00fcr.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"frequently-asked-questions-faq\">S\u0131k\u00e7a Sorulan Sorular (SSS)<\/h2>\n\n\n<div id=\"rank-math-faq\" class=\"rank-math-block\">\n<div class=\"rank-math-list\">\n<div id=\"faq-question-1776705232498\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">CK nedir ve Duchenne gen terapisinde neden \u00f6nemlidir?<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>Kreatin Kinaz (CK), kas lifleri hasar g\u00f6rd\u00fc\u011f\u00fcnde kan dola\u015f\u0131m\u0131na sal\u0131nan bir enzimdir. Duchenne kas distrofisinde, kaslar s\u00fcrekli olarak par\u00e7aland\u0131\u011f\u0131 i\u00e7in CK seviyeleri genellikle \u00e7ok y\u00fcksektir.<\/p>\n<p>Gen terapisi sonras\u0131nda CK&#039;n\u0131n \u00f6nemi \u015fu nedenlerden kaynaklanmaktad\u0131r:<\/p>\n<p>\u2022 CK seviyesindeki d\u00fc\u015f\u00fc\u015f, kas hasar\u0131n\u0131n azald\u0131\u011f\u0131n\u0131 g\u00f6sterebilir.<br \/>\u2022 Stabil veya y\u00fckselen CK seviyesi, hastal\u0131\u011f\u0131n devam etti\u011fini g\u00f6sterebilir.<br \/>\u2022 CK e\u011filimleri, tedavinin yaln\u0131zca g\u00f6r\u00fcn\u00fcr bir etkiye sahip olup olmad\u0131\u011f\u0131n\u0131 de\u011fil, biyolojik bir etkiye sahip olup olmad\u0131\u011f\u0131n\u0131 de\u011ferlendirmeye yard\u0131mc\u0131 olur.<\/p>\n<p>K\u0131sacas\u0131: <strong>CK, videolar\u0131n tek ba\u015f\u0131na sa\u011flayamayaca\u011f\u0131 objektif biyokimyasal kan\u0131tlar sunar.<\/strong><\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1776705321179\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">Gen terapisi sonras\u0131 iyile\u015fme videolar\u0131 g\u00fcvenilir bir kan\u0131t m\u0131d\u0131r?<\/h3>\n<div class=\"rank-math-answer\">\n\n<p><strong>Hay\u0131r, videolar g\u00fcvenilir bilimsel kan\u0131t de\u011fildir.<\/strong><\/p>\n<p>Ger\u00e7ek anlar\u0131 yans\u0131t\u0131yor olsalar da, \u015fu eksiklikleri var:<\/p>\n<p>\u2022 Standartla\u015ft\u0131r\u0131lm\u0131\u015f test ko\u015fullar\u0131<br \/>\u2022 Temel kar\u015f\u0131la\u015ft\u0131rmalar<br \/>\u2022 Uzun s\u00fcreli takip<br \/>\u2022 Nesnel \u00f6l\u00e7\u00fcm \u00f6l\u00e7ekleri<\/p>\n<p>Videoda daha g\u00fc\u00e7l\u00fc g\u00f6r\u00fcnen bir \u00e7ocu\u011fun etkilenmesinde \u015fu fakt\u00f6rler etkili olabilir:<\/p>\n<p>\u2022 Motivasyon veya te\u015fvik<br \/>\u2022 Se\u00e7ici kay\u0131t veya d\u00fczenleme<br \/>\u2022 G\u00fcnl\u00fck do\u011fal de\u011fi\u015fim<\/p>\n<p>Doktorlar klinik de\u011ferlendirme i\u00e7in \u015funlara g\u00fcvenirler:<\/p>\n<p>\u2022 Fonksiyonel \u00f6l\u00e7ekler (\u00f6rn. NSAA)<br \/>\u2022 CK, AST, ALT gibi biyobelirte\u00e7ler<br \/>\u2022 Kontroll\u00fc klinik \u00e7al\u0131\u015fma verileri<\/p>\n<p><strong>Videolar, tedavinin etkinli\u011fini g\u00f6sterebilir ancak do\u011frulayamaz.<\/strong><\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1776705412246\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">Gen terapisi sonras\u0131nda AST ve ALT d\u00fczeyleri neden rapor edilmelidir?<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>AST (Aspartat Aminotransferaz) ve ALT (Alanin Aminotransferaz), hem karaci\u011fer hem de kas sa\u011fl\u0131\u011f\u0131n\u0131 yans\u0131tan enzimlerdir.<\/p>\n<p>Gen terapisi sonras\u0131nda kritik \u00f6neme sahip olmalar\u0131n\u0131n nedenleri \u015funlard\u0131r:<\/p>\n<p>\u2022 Y\u00fcksek seviyeler karaci\u011fer stresi veya toksisitesine i\u015faret edebilir.<br \/>\u2022 DMD&#039;de bunlar ayn\u0131 zamanda kas y\u0131k\u0131m\u0131n\u0131 da yans\u0131t\u0131r.<br \/>\u2022 Ani y\u00fckseli\u015fler g\u00fcvenlik endi\u015felerine i\u015faret edebilir.<\/p>\n<p>AST ve ALT&#039;yi izlemek \u015fu konularda yard\u0131mc\u0131 olur:<\/p>\n<p>\u2022 Tedavinin g\u00fcvenli\u011fini de\u011ferlendirmek<br \/>\u2022 Olumsuz etkileri erken tespit etmek<br \/>\u2022 V\u00fccudun g\u00f6zle g\u00f6r\u00fcl\u00fcr de\u011fi\u015fikliklerin \u00f6tesinde nas\u0131l tepki verdi\u011fini anlamak.<\/p>\n<p><strong>AST ve ALT verileri olmadan g\u00fcvenlik de\u011ferlendirmesi eksik kal\u0131r.<\/strong><\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1776705562117\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">\u0130la\u00e7 \u015firketleri neden biyobelirte\u00e7 verilerinin tamam\u0131n\u0131 kamuoyuyla payla\u015fm\u0131yor?<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>Tek bir neden yok, ancak yayg\u0131n fakt\u00f6rler \u015funlard\u0131r:<\/p>\n<p>\u2022 Yasal s\u00fcre\u00e7 takvimleri (inceleme s\u00fcre\u00e7lerinden sonra yay\u0131nlanan veriler)<br \/>\u2022 Se\u00e7ici haber stratejileri (\u00f6ncelikle olumlu sonu\u00e7lar\u0131 vurgulama)<br \/>\u2022 Ham verilerin yanl\u0131\u015f yorumlanmas\u0131na ili\u015fkin endi\u015feler<br \/>\u2022 Rekabet\u00e7i ve ticari hususlar<\/p>\n<p>Ancak gen terapisi gibi y\u00fcksek riskli tedavilerde:<\/p>\n<p>\u2022 \u015eeffafl\u0131k bu endi\u015felerin \u00f6n\u00fcne ge\u00e7melidir.<br \/>\u2022 Hastalar ve aileleri k\u0131smi de\u011fil, eksiksiz bilgiye ihtiya\u00e7 duyarlar.<\/p>\n<p>Avrupa \u0130la\u00e7 Ajans\u0131 gibi kurulu\u015flardan, biyobelirte\u00e7ler de dahil olmak \u00fczere g\u00fcvenlik ve etkinlik verilerinin eri\u015filebilir ve g\u00fcvenilir olmas\u0131n\u0131 sa\u011flamalar\u0131 beklenmektedir.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1776705628252\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">Gen terapisi de\u011ferlendirilirken aileler videolar\u0131n yerine nelere dikkat etmelidir?<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>Aileler, a\u015fa\u011f\u0131dakiler de dahil olmak \u00fczere, objektif ve klinik olarak do\u011frulanm\u0131\u015f verilere \u00f6ncelik vermelidir:<\/p>\n<p>\u2022 Tedavi \u00f6ncesi ve sonras\u0131 CK d\u00fczeyi e\u011filimleri<br \/>\u2022 G\u00fcvenlik izleme i\u00e7in AST ve ALT de\u011ferleri<br \/>\u2022 Kontroll\u00fc klinik ara\u015ft\u0131rmalardan elde edilen sonu\u00e7lar<br \/>\u2022 Fonksiyonel de\u011ferlendirmeler (\u00f6rne\u011fin, <a href=\"https:\/\/dmdwarrior.com\/tr\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" data-type=\"post\" data-id=\"4959\" rel=\"noreferrer noopener\">NSAA puanlar\u0131<\/a>)<br \/>\u2022 Uzun s\u00fcreli takip verileri (12-24 ay veya daha fazla)<\/p>\n<p>Ayr\u0131ca aileler \u015funlar\u0131 yapmal\u0131d\u0131r:<\/p>\n<p>\u2022 N\u00f6rom\u00fcsk\u00fcler uzmanlara dan\u0131\u015fmak<br \/>\u2022 Avrupa \u0130la\u00e7 Ajans\u0131 gibi g\u00fcvenilir d\u00fczenleyici kurulu\u015flardan gelen verileri incelemek<br \/>\u2022 Yaln\u0131zca sosyal medyaya dayanarak var\u0131lan sonu\u00e7lara kar\u015f\u0131 temkinli olmak<\/p>\n<p><strong>Temel ilke: Kararlar, tekil g\u00f6rsel izlenimlere de\u011fil, verilere dayanmal\u0131d\u0131r.<\/strong><\/p>\n\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n\n\n<p class=\"wp-block-paragraph\">Devam\u0131n\u0131 oku: <a href=\"https:\/\/dmdwarrior.com\/tr\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Kreatin Kinaz (CK), AST, ALT ve Distrofin D\u00fczeylerinin Neden A\u00e7\u0131klanmas\u0131 Gerekir?<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"final-thoughts\">Son D\u00fc\u015f\u00fcnceler<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne kas distrofisinde gen tedavisinin gelece\u011fi tek bir prensibe ba\u011fl\u0131d\u0131r: <strong>Kan\u0131t, video payla\u015f\u0131mlar\u0131ndan \u00f6nce gelmelidir.<\/strong>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Solid Biosciences (SGT-003), REGENXBIO (RGX-202), Genethon (GNT0004) ve Satellos (SAT-3247) dahil olmak \u00fczere tedaviler geli\u015ftiren \u015firketlerin, Duchenne gen terapisi sonras\u0131nda CK d\u00fczeylerini, AST ve ALT verileriyle birlikte, Avrupa \u0130la\u00e7 Ajans\u0131 gibi resmi kanallar arac\u0131l\u0131\u011f\u0131yla kamuoyuna a\u00e7\u0131klamalar\u0131 bekleniyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Toplulu\u011fun daha fazla videoya ihtiyac\u0131 yok.<br><strong>\u00d6l\u00e7\u00fclebilir, do\u011frulanabilir ve g\u00fcvenilir verilere ihtiya\u00e7 duyuyor.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu standart kar\u015f\u0131lanana kadar \u015f\u00fcphecilik direni\u015f de\u011fil, sorumluluktur.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"stop-the-noise-just-share-the-data-for-duchenne-muscular-dystrophy\"><strong>Gereksiz ve bilimsel olmayan videolara g\u00fcvenmeyi b\u0131rak\u0131n; Duchenne kas distrofisiyle ilgili verileri payla\u015fmaya odaklan\u0131n.<\/strong><\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Devam\u0131n\u0131 oku: <a href=\"https:\/\/dmdwarrior.com\/tr\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">NSAA Tek Ba\u015f\u0131na Yeterli De\u011fil: Kreatin Kinaz (CK), AST, ALT ve Distrofin D\u00fczeylerinin A\u00e7\u0131klanmas\u0131 Neden \u00d6nemli?<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"academic-sources-and-references\"><strong>Akademik Kaynaklar ve Referanslar<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bushby, K., Finkel, R., Birnkrant, DJ ve di\u011ferleri.<\/strong> (2010). Duchenne kas distrofisinin tan\u0131 ve y\u00f6netimi, b\u00f6l\u00fcm 1: tan\u0131 ve farmakolojik ve psikososyal y\u00f6netim. The Lancet Neurology, 9(1), 77\u201393. <a href=\"https:\/\/doi.org\/10.1016\/S1474-4422(09)70271-6\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1016\/S1474-4422(09)70271-6<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bushby, K., Finkel, R., Birnkrant, DJ ve di\u011ferleri.<\/strong> (2010). Duchenne kas distrofisinin tan\u0131 ve y\u00f6netimi, b\u00f6l\u00fcm 2: multidisipliner bak\u0131m\u0131n uygulanmas\u0131. The Lancet Neurology, 9(2), 177\u2013189. <a href=\"https:\/\/doi.org\/10.1016\/S1474-4422(09)70272-8\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1016\/S1474-4422(09)70272-8<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Birnkrant, DJ, Bushby, K., Bann, CM, ve di\u011ferleri.<\/strong> (2018). Duchenne kas distrofisinin tan\u0131 ve y\u00f6netimi, b\u00f6l\u00fcm 1: tan\u0131, n\u00f6rom\u00fcsk\u00fcler, rehabilitasyon, endokrin ve gastrointestinal y\u00f6netim. The Lancet Neurology, 17(3), 251\u2013267. <a href=\"https:\/\/doi.org\/10.1016\/S1474-4422(18)30024-3\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1016\/S1474-4422(18)30024-3<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Birnkrant, DJ, Bushby, K., Bann, CM, ve di\u011ferleri.<\/strong> (2018). Duchenne kas distrofisinin tan\u0131 ve y\u00f6netimi, b\u00f6l\u00fcm 2: solunum, kalp, kemik sa\u011fl\u0131\u011f\u0131 ve ortopedik y\u00f6netim. The Lancet Neurology, 17(4), 347\u2013361. <a href=\"https:\/\/doi.org\/10.1016\/S1474-4422(18)30025-5\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1016\/S1474-4422(18)30025-5<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Mendell, JR, \u015eahenk, Z., Lehman, K., ve di\u011ferleri.<\/strong> (2020). Duchenne kas distrofisi olan \u00e7ocuklarda rAAVrh74.MHCK7.mikro-distrofinin sistemik da\u011f\u0131t\u0131m\u0131n\u0131n de\u011ferlendirilmesi. New England Journal of Medicine, 383(21), 2039\u20132047. <a href=\"https:\/\/doi.org\/10.1056\/NEJMoa2002295\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1056\/NEJMoa2002295<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Duan, D.<\/strong> (2018). Duchenne kas distrofisi i\u00e7in sistemik AAV mikro-distrofin gen terapisi. Molek\u00fcler Terapi, 26(10), 2337\u20132356. <a href=\"https:\/\/doi.org\/10.1016\/j.ymthe.2018.03.012\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1016\/j.ymthe.2018.03.012<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Hathout, Y., Brody, E., Clemens, PR ve di\u011ferleri.<\/strong> (2015). Duchenne kas distrofisinde b\u00fcy\u00fck \u00f6l\u00e7ekli serum protein biyobelirte\u00e7 ke\u015ffi. Ulusal Bilimler Akademisi Bildirileri (PNAS), 112(23), 7153\u20137158. <a href=\"https:\/\/doi.org\/10.1073\/pnas.1507719112\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1073\/pnas.1507719112<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>McDonald, CM ve di\u011ferleri.<\/strong> (2018). Duchenne kas distrofisinde serum biyobelirte\u00e7leri. N\u00f6roloji, 90(21), e1839\u2013e1848. <a href=\"https:\/\/doi.org\/10.1212\/WNL.0000000000005515\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1212\/WNL.0000000000005515<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Brancaccio, P., Lippi, G. ve Maffulli, N.<\/strong> (2007). Kas hasar\u0131n\u0131n biyokimyasal belirte\u00e7leri. Klinik Kimya ve Laboratuvar T\u0131bb\u0131, 45(5), 603\u2013611. <a href=\"https:\/\/doi.org\/10.1515\/CCLM.2007.115\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1515\/CCLM.2007.115<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nathwani, RA, Pais, S., Reynolds, TB ve Kaplowitz, N.<\/strong> (2005). \u0130skelet kas\u0131 hastal\u0131klar\u0131nda serum alanin aminotransferaz. Hepatoloji, 41(2), 380\u2013382. <a href=\"https:\/\/doi.org\/10.1002\/hep.20548\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1002\/hep.20548<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>High, KA ve Roncarolo, MG.<\/strong> (2019). Gen terapisi. New England T\u0131p Dergisi, 381(5), 455\u2013464. <a href=\"https:\/\/doi.org\/10.1056\/NEJMra1706910\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1056\/NEJMra1706910<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Mendell, JR ve Rodino-Klapac, LR.<\/strong> (2020). Duchenne kas distrofisi: CRISPR\/Cas9 tedavisi. H\u00fccre Ara\u015ft\u0131rmas\u0131, 30(6), 465\u2013466. <a href=\"https:\/\/doi.org\/10.1038\/s41422-020-0317-7\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1038\/s41422-020-0317-7<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Avrupa \u0130la\u00e7 Ajans\u0131 (EMA).<\/strong> Gen terapisi d\u00fczenleyici k\u0131lavuzlar\u0131 ve de\u011ferlendirme raporlar\u0131. \u015eu adresten ula\u015f\u0131labilir: <a href=\"https:\/\/www.ema.europa.eu\" target=\"_blank\" rel=\"noopener\">https:\/\/www.ema.europa.eu<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>ABD G\u0131da ve \u0130la\u00e7 \u0130daresi (FDA).<\/strong> End\u00fcstri i\u00e7in insan gen terapisi k\u0131lavuzu. \u015eurada mevcuttur: <a href=\"https:\/\/www.fda.gov\" target=\"_blank\" rel=\"noopener\">https:\/\/www.fda.gov<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>In the evolving landscape of gene therapy for Duchenne muscular dystrophy (DMD), one issue is becoming increasingly clear: CK levels after Duchenne gene therapy are far more important than viral videos circulating online. Families are being exposed to emotionally powerful, time-lapse videos that appear to show improvement after treatment. However, these visual narratives are often [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":7673,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[697,698,696,701,387,522,75,700,68,699,523,61],"class_list":["post-7667","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-alanine-aminotransferase","tag-alt","tag-aspartate-aminotransferase","tag-ast-alt-biomarkers","tag-astrazeneca","tag-biomarkers","tag-ck","tag-ck-levels-duchenne","tag-creatine-kinase","tag-dmd-biomarkers","tag-duchenne-biomarkers","tag-gene-therapy"],"subtitle":"Duchenne gen terapisi sonras\u0131 CK d\u00fczeyleri, videolar\u0131n g\u00f6steremedi\u011fi \u015feyleri ortaya koyuyor. Duchenne kas distrofisinde, sonu\u00e7lar\u0131 viral videolar de\u011fil, biyobelirte\u00e7 \u015feffafl\u0131\u011f\u0131 belirlemelidir. CK, AST ve ALT verileri neden hala gizleniyor? Duygular yerine kan\u0131t talep etmenin zaman\u0131 geldi.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7667","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=7667"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7667\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/7673"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=7667"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=7667"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=7667"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}