{"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"],"_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}]}}