{"id":4096,"date":"2025-07-15T18:08:06","date_gmt":"2025-07-15T15:08:06","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4096"},"modified":"2025-07-15T18:11:40","modified_gmt":"2025-07-15T15:11:40","slug":"medera-cardiomyopathy-gene-therapy-trial-srd-001-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/medera-cardiomyopathy-gene-therapy-trial-srd-001-for-duchenne-muscular-dystrophy\/","title":{"rendered":"Medera ve Kansas \u00dcniversitesi T\u0131p Merkezi, Duchenne Musk\u00fcler Distrofi \u0130li\u015fkili Kardiyomiyopati \u0130\u00e7in \u00d6nc\u00fc Gen Terapisi Denemesinde (SRD-001) \u0130lk Hastan\u0131n Tedavi Edildi\u011fini Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Yeni nesil tedaviler geli\u015ftirerek kardiyovask\u00fcler hastal\u0131klar\u0131 hedeflemeye odaklanan klinik a\u015famadaki bir biyofarmas\u00f6tik \u015firketi olan Medera ve klinik geli\u015ftirme b\u00f6l\u00fcm\u00fc Sardocor, Kansas \u00dcniversitesi T\u0131p Merkezi ile birlikte bug\u00fcn MUSIC-DMD Faz 1b klinik denemesinde ilk hastan\u0131n ba\u015far\u0131l\u0131 bir \u015fekilde tedavi edildi\u011fini duyurdu. Deneme, Duchenne kas distrofisine (DMD-CM) ba\u011fl\u0131 kardiyomiyopati i\u00e7in tek seferlik bir gen tedavisi olan AAV1.SERCA2a&#039;y\u0131 ara\u015ft\u0131r\u0131yor.<\/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=\"#first-patient-treated\">SRD-001 ile Tedavi Edilen \u0130lk Hasta<\/a><\/li><li><a href=\"#gene-therapy-for-dmd-cardiomyopathy-srd-003\">DMD Kardiyomiyopatisi i\u00e7in Gen Terapisi: SRD-001<\/a><\/li><li><a href=\"#music-dmd-phase-1-b-trial\">MUSIC-DMD Faz 1b Denemesi<\/a><\/li><li><a href=\"#bbm-d-101-clinical-trial-nct-07058662\">SRD-001 Klinik Ara\u015ft\u0131rmas\u0131 \u2013 NCT06224660<\/a><\/li><li><a href=\"#bbm-d-101-phase-1-2-participation-criteria\">SRD-001 A\u015fama 1b Kat\u0131l\u0131m Kriterleri<\/a><\/li><li><a href=\"#how-can-i-contact-with-belief-bio-med\">Sardocor ile Nas\u0131l \u0130leti\u015fime Ge\u00e7ebilirim?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"first-patient-treated\">SRD-001 ile Tedavi Edilen \u0130lk Hasta<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Hasta, Medera'n\u0131n tescilli minimal invaziv intrakoroner inf\u00fczyon metodolojisi kullan\u0131larak sol radyal arter yoluyla ba\u015far\u0131yla tedavi edildi. Hasta i\u015flemi iyi tolere etti ve g\u00f6zlem i\u00e7in bir gece hastanede kald\u0131ktan sonra taburcu edildi.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"gene-therapy-for-dmd-cardiomyopathy-srd-003\">DMD Kardiyomiyopatisi i\u00e7in Gen Terapisi: SRD-001<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Medera&#039;nin CEO&#039;su ve kurucu orta\u011f\u0131 Dr. Ronald Li, &quot;Bu d\u00f6n\u00fcm noktas\u0131, DMD ile ili\u015fkili kardiyomiyopatinin tedavisinde \u00f6nemli bir ilerlemeyi temsil ediyor,&quot; dedi. &quot;Solunum m\u00fcdahaleleri sayesinde sa\u011fkal\u0131m oranlar\u0131 iyile\u015ftik\u00e7e, kardiyak komplikasyonlar DMD hastalar\u0131nda \u00f6nde gelen \u00f6l\u00fcm nedeni haline geldi. Sardocor b\u00f6l\u00fcm\u00fcm\u00fcz\u00fcn yenilik\u00e7i, insanlarda ilk gen tedavisi, di\u011fer endikasyonlarda terap\u00f6tik geni do\u011frudan etkilenen kalp dokusuna g\u00fcvenli ve g\u00fcvenilir bir \u015fekilde iletti\u011fi g\u00f6sterilen tescilli intrakoroner inf\u00fczyon metodolojimiz arac\u0131l\u0131\u011f\u0131yla, bu kritik kar\u015f\u0131lanmam\u0131\u015f t\u0131bbi ihtiyaca y\u00f6nelik umut verici bir \u00e7\u00f6z\u00fcm sunuyor.&quot;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"music-dmd-phase-1-b-trial\">MUSIC-DMD Faz 1b Denemesi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">MUSIC-DMD Faz 1b \u00e7al\u0131\u015fmas\u0131, DMD ile ili\u015fkili kardiyomiyopatisi olan yeti\u015fkin erkeklerde SERCA2a genini do\u011frudan kalp kas\u0131 h\u00fccrelerine ileten adeno-ili\u015fkili vir\u00fcs serotipi 1 (AAV1) gen terapisi olan AAV1.SERCA2a&#039;n\u0131n g\u00fcvenli\u011fini ve etkinli\u011fini de\u011ferlendiren a\u00e7\u0131k etiketli, kontroll\u00fc bir \u00e7al\u0131\u015fmad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fmaya d\u00fc\u015f\u00fck doz, y\u00fcksek doz ve kontrol gruplar\u0131ndan en fazla 12 kat\u0131l\u0131mc\u0131 kat\u0131lacakt\u0131r. DMD ile ili\u015fkili kardiyomiyopati, kalp kas\u0131 h\u00fccrelerinde kalsiyum a\u015f\u0131r\u0131 y\u00fcklenmesi ile karakterize olup ilerleyici fibroz ve kalp yetmezli\u011fine yol a\u00e7ar. AAV1.SERCA2a, SERCA2a protein \u00fcretimini art\u0131rarak DMD-CM hastalar\u0131nda kalsiyum kullan\u0131m\u0131n\u0131 eski haline getirmeyi ve b\u00f6ylece altta yatan hastal\u0131k s\u00fcrecini tersine \u00e7evirmeyi ama\u00e7lamaktad\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"bbm-d-101-clinical-trial-nct-07058662\">SRD-001 Klinik Ara\u015ft\u0131rmas\u0131 \u2013 NCT06224660<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fma Ba\u015flang\u0131c\u0131 (Ger\u00e7ek)<\/strong>: 2024-10-02<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Birincil Tamamlanma (Tahmini)<\/strong>: 2027-10<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fman\u0131n Tamamlanmas\u0131 (Tahmini)<\/strong>: 2030-10<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Kat\u0131l\u0131mc\u0131 Say\u0131s\u0131 (Tahmini)<\/strong>: 12 (Erkek)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Konumlar<\/strong>: ABD<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fmaya Uygun Ya\u015flar<\/strong>: 18 Ya\u015f ve \u00fczeri (Yeti\u015fkin, Ya\u015fl\u0131 Yeti\u015fkin)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"bbm-d-101-phase-1-2-participation-criteria\">SRD-001 A\u015fama 1b Kat\u0131l\u0131m Kriterleri<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Uygun olabilmek i\u00e7in ki\u015filerin dahil olma gerekliliklerini yerine getirmeleri gerekir. Bir ki\u015finin ba\u015fka bir rahats\u0131zl\u0131\u011f\u0131 varsa veya gen terapisi almas\u0131n\u0131 engelleyebilecek bir tedavi g\u00f6r\u00fcyorsa, kat\u0131l\u0131m g\u00f6steremeyebilir. Dahil olma ve hari\u00e7 tutma kriterlerinin tamam\u0131na bu \u00e7al\u0131\u015fma ba\u011flant\u0131s\u0131na t\u0131klayarak ula\u015f\u0131labilir \u2013 <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT06224660\" target=\"_blank\" rel=\"noreferrer noopener\">NCT06224660<\/a> \u2013.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-can-i-contact-with-belief-bio-med\">Sardocor ile Nas\u0131l \u0130leti\u015fime Ge\u00e7ebilirim?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Ad\u0131: Sardocor Corp.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Telefon Numaras\u0131: +1-617-880-7616<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">E-posta: info@sardocorcorp.com<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Medera a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing next-generation therapeutics, and its clinical development division Sardocor, together with the University of Kansas Medical Center, today announced the successful treatment of the first patient in the MUSIC-DMD Phase 1b clinical trial. The trial is investigating AAV1.SERCA2a, a one-time gene therapy treatment for [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4103,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[89,362,391,390,392,393],"class_list":["post-4096","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-cardiomyopathy","tag-dmd-cardiomyopathy","tag-kansas-medical-center","tag-medera","tag-music-dmd","tag-sardocor"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4096","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=4096"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4096\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4103"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4096"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4096"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4096"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}