{"id":3244,"date":"2025-03-18T13:38:40","date_gmt":"2025-03-18T10:38:40","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3244"},"modified":"2025-03-18T13:38:42","modified_gmt":"2025-03-18T10:38:42","slug":"significant-improvements-seen-in-3-children-receiving-dystrogen-therapeutics-dt-dec01-cell-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/de\/significant-improvements-seen-in-3-children-receiving-dystrogen-therapeutics-dt-dec01-cell-therapy\/","title":{"rendered":"Signifikante Verbesserungen bei 3 Kindern, die die Dystrogen Therapeutics&#039; DT-DEC01-Zelltherapie erhalten"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\">In non-ambulatory individuals with Duchenne muscular dystrophy (DMD), DT-DEC01 (Dystrogen Therapeutics), an experimental dystrophin expressing chimeric (DEC) cell treatment, was found to be safe over a 24-month period in a pilot single-site, open-label research. The results further support the development of the drug as a treatment for DMD, independent of gene mutation or disease progression, when combined with advancements on functional tests.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Under the direction of Maria Siemionow, MD, PhD, DSc, professor and director of Microsurgery Research at the University of Illinois, the analysis included 24-month data on three individuals, without immunosuppression, ages 11\u201316, who received dosages per kilogram body weight, respectively.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Dystrogen Therapeutics\u2019 Chimeric Cells<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Treatment with the cell therapy causes no significant adverse events (AEs), donor-specific antibodies (DSA), or study-related AEs for up to 24 months, according to a presentation made at the 2025 Muscular Dystrophy Association (MDA) Clinical &amp; Scientific meeting, which took place in Dallas, Texas, from March 16\u201319. [<a href=\"https:\/\/dystrogen.com\/chimeric-cells\/\" target=\"_blank\" rel=\"noreferrer noopener\">Chimeric Cells<\/a>]<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Effects of DT-DEC01 Cell Therapy on Different Exons<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Patient 1, a 15-year-old with exon 48-50 deletion, showed improvements in echocardiography EF by 12%, arm movements by 9%, and Motor Unit Potentials (MUP) duration, both in deltoideus (53%) and biceps brachii (23%) at 18 months. In addition, this patient saw enhancements in PUL 2.0 test score by 5%, grip strength by 6%, and spirometry by 17% after 24 months since the original procedure.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Patient 2, an 11-year-old with exon 52 deletion, demonstrated a 17% improvement in echocardiography EF at 12 months, along with a 5% increase in PUL 2.0 test score and enhancements in MUP duration, including 19% in the deltoideus and 51% in the biceps brachii, at 18 months. Furthermore, this patient experienced a 59% improvement in spirometry and a remarkable 1150% increase in arm movements at 24 months.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Patient 3, a 16-year-old with a nonsense mutation, reported improvements at 12 months post-DT-DEC01 administration, including a 6% increase in PUL 2.0 test score, a 34% improvement in grip strength, an 11% enhancement in echocardiography EF, and increases in MUP duration, with 49% in the deltoideus and 29% in the biceps brachii.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">Conclusion<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">In the 12-month published data, the study authors wrote that the findings &#8220;supports the potential benefits of DT-DEC01 therapy in improving cardiac, respiratory and skeletal muscle function in patients with DMD after systemic-intraosseous administration, providing hope for better outcomes and enhanced quality of life for DMD patients. Additionally, this study also highlights use of EMG as a valuable biomarker for monitoring functional changes in muscles affected by DMD after DT-DEC01 therapy.&#8221;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>In non-ambulatory individuals with Duchenne muscular dystrophy (DMD), DT-DEC01 (Dystrogen Therapeutics), an experimental dystrophin expressing chimeric (DEC) cell treatment, was found to be safe over a 24-month period in a pilot single-site, open-label research. The results further support the development of the drug as a treatment for DMD, independent of gene mutation or disease progression, [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3249,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[303,304,306,305,302],"class_list":["post-3244","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-cell-therapy","tag-chimeric-cell-therapy","tag-chimeric-cells","tag-dt-dec01","tag-dystrogen-therapeutics"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/posts\/3244","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/comments?post=3244"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/posts\/3244\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/media\/3249"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/media?parent=3244"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/categories?post=3244"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/tags?post=3244"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}