{"id":1495,"date":"2026-11-01T11:26:14","date_gmt":"2026-11-01T11:26:14","guid":{"rendered":"https:\/\/healthdailyusa.com\/?page_id=1495"},"modified":"2026-11-01T17:45:13","modified_gmt":"2026-11-01T17:45:13","slug":"gene-therapy-trial-for-duchenne-muscular-dystrophy-begins","status":"publish","type":"page","link":"https:\/\/healthdailyusa.com\/index.php?page_id=1495","title":{"rendered":"HealthDailyUSA.com &#8211; Gene Therapy Trial for Duchenne Muscular Dystrophy Begins"},"content":{"rendered":"\n<div class=\"wp-block-group custom is-layout-flow wp-block-group-is-layout-flow\"><\/div>\n\n\n<div id=\"fb-root\" class=\"wp-block-group is-layout-flow wp-block-group-is-layout-flow\"><\/div>\n\n\n<div style=\"display: none\">\n  <div id=\"inline1\" style=\"color: #004E98;text-align:center;font: 24px\/32px Calibri;margin-bottom: 10px;line-height:27px;overflow: hidden;width: 490px;height: 270px;padding-left: 8px;margin: auto\">\n    <h1 style=\"font: 48px Calibri\">Thank You!<\/h1>\n    <hr>    \n    You have made a smart decision! 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id=\"system-message-container\">\n<\/div>\n        \n<!-- Start K2 Item Layout -->\n<span id=\"startOfPageId1170\"><\/span>\n\n<div id=\"k2Container\" class=\"itemView\">\n\n\t<!-- Plugins: BeforeDisplay -->\n\t\n\t<!-- K2 Plugins: K2BeforeDisplay -->\n\t\n\t<div class=\"itemHeader\">\n\n\t\t\t\t<!-- Date created -->\n\t\t<span class=\"itemDateCreated\">\n\t\t\tFriday, 07 April 2006 00:00\t\t<\/span>\n\t\t\n\t  \t  <!-- Item title -->\n\t  <h1 class=\"itemTitle\">\n\t\t\t\t\t\t\t\t\n\t\t\tGene Therapy Trial for Duchenne \t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"cunudnxuzbfjfxemdzpj\"><\/span>\n\n\nMuscular Dystrophy Begins\n\t\t\t\n\t  <\/h1>\n\t  \n\t\t\n  <\/div>\n\n  <!-- Plugins: AfterDisplayTitle -->\n  \n  <!-- K2 Plugins: K2AfterDisplayTitle -->\n  \n\t  <div class=\"itemToolbar\">\n\t\t<ul>\n\t\t\t\n\t\t\t\n\t\t\t\n\t\t\t\t\t\t<!-- Item Social Button -->\n\t\t\t<li>\n\t\t\t\t\n\n\n<!-- AddThis Button BEGIN -->\n<div class=\"addthis_toolbox addthis_default_style\" style=\"margin-top: 15px\">\n\n\n\n\n\n<\/div>\n<div style=\"position: absolute;margin-bottom: 1px;margin-top: -19px;margin-left: 164px\">\n\t\t\t\n\t\t\t&lt;div class=&quot;fb-like&quot; data-send=&quot;false&quot; data-layout=&quot;button_count&quot; data-width=&quot;200&quot; data-show-faces=&quot;true&quot; data-font=&quot;verdana&quot; \n<\/div>\n<div style=\"position: absolute;margin-bottom: 1px;margin-top: -20px;margin-left: 176px\">\nTweet\n<\/div>\n<\/div>\n\n<!-- AddThis Button END -->\n\n\n\n    \n\n\n\t\t\t<\/li>\n\t\t\t\n\t\t\t\n\t\t\t\n\t\t\t\t\t<\/ul>\n\t\t<div class=\"clr\"><\/div>\n  <\/div>\n\t\n\t\n  <div class=\"itemBody\">\n\n\t  <!-- Plugins: BeforeDisplayContent -->\n\t  \n\t  <!-- K2 Plugins: K2BeforeDisplayContent -->\n\t  \n\t  \n\t  \t  <!-- Item text -->\n\t  <div class=\"itemFullText\">\n\t  \t<p>FRIDAY, April 7 (HealthDay News) &#8212; The first U.S. gene therapy trial focused on Duchenne muscular dystrophy is under way at Columbus Children&#8217;s Hospital in Columbus, Ohio.<\/p>\n\n<p>&#8220;This\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"cunudnxuzbfjfxemdzpj\"><\/span>\n\n is a long-awaited step in the overall plan to help kids with muscular dystrophy, especially for those of us who have been involved in clinical trials for many years,&#8221; neurologist Dr. Jerry Mendell, co-director of the Muscular Dystrophy Association Clinic at Columbus Children&#8217;s Hospital, said in a prepared statement. &#8220;We are all hopeful that this effort will help guide us toward clinically meaningful results as we move forward with this form of treatment.&#8221; <\/p>\n\n<p>The study will assess the safety and efficacy of a functional gene for the muscle protein dystrophin. Duchenne muscular dystrophy occurs when a gene on the X chromosome fails to make this essential muscle protein. The disease begins in early childhood and causes progressive loss of muscle strength and bulk. People with the disease usually die in their 20s from respiratory or cardiac muscle failure.<\/p>\n\n<p>Six\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"cunudnxuzbfjfxemdzpj\"><\/span>\n\n Duchenne-affected boys will take part in the study. Each boy will receive an injection of dystrophin genes in the muscle of one arm and a placebo in the other arm. Neither the boys nor the researchers will know which arm received the genes.<\/p>\n\n<p>After\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"cunudnxuzbfjfxemdzpj\"><\/span>\n\n several weeks, the researchers will analyze samples of muscle tissue from both arms of all the study participants, who will also undergo strength and health testing. The researchers will assess whether this gene therapy is likely to result in persistent production of dystrophin in muscle cells.<\/p>\n\n<p>More information<\/p>\n\n<p>The\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"cunudnxuzbfjfxemdzpj\"><\/span>\n\n Muscular Dystrophy Association has more about Duchenne Muscular Dystrophy.<\/p>\n\t  <\/div>\n\t  \n\t\t<div class=\"clr\"><\/div>\n\n\t  \n\t\t\n\t  <!-- Plugins: AfterDisplayContent -->\n\t  \n\t  <!-- K2 Plugins: K2AfterDisplayContent -->\n\t  \n\t  <div class=\"clr\"><\/div>\n  <\/div>\n\n\t\n  \n  \n  \n\t\t\t\n  \n\t<div class=\"clr\"><\/div>\n\n  \n  \n  \n  <!-- Plugins: AfterDisplay -->\n  \n  <!-- K2 Plugins: K2AfterDisplay -->\n  \n  \n \n\t\t<div class=\"itemBackToTop\">\n\t\t<a class=\"k2Anchor\" href=\"https:\/\/healthdailyusa.com\/index.php?page_id=1495#startOfPageId1170\">\n\t\t\tback to top\t\t<\/a>\n\t<\/div>\n\t\n\t<div class=\"clr\"><\/div>\n<\/div>\n<!-- End K2 Item Layout -->\n\n<!-- JoomlaWorks \"K2\" (v2.5.4) | Learn more about K2 at http:\/\/getk2.org -->\n\n\n              \n                <div class=\"artBanner\">  \n            <div class=\"carouselbanner\">\n                <div id=\"wrap\">\n                    <ul id=\"mycarousel90\" class=\"jcarousel-skin-tango\">\n                        <li>\n                                                            <!-- Conversant Media 160x600 WIDE SKYSCRAPER CODE for AdRx - PD ROH -->\n                                \n                                \n                                \n                                <!-- Conversant Media 160x600 WIDE SKYSCRAPER CODE for AdRx - PD ROH -->\n                                                   \n                      <\/li>\n                    <\/ul>\n                <\/div>\n            <\/div>\n        <\/div>      \n          <\/div>\n        <div class=\"clr\"><\/div>    \n    \n       <div id=\"bottombar\">\n        <div class=\"panel\" style=\"float:none !important\">\n            <div class=\"bannergroup\">\n                <div class=\"banneritem\">\n                                        \t<!-- Conversant Media 300x250 Medium Rectangle CODE for AdRx - PD ROH -->\n\t\t\t\t\t\t\n                        \n                        \n                        <!-- Conversant Media 300x250 Medium Rectangle CODE for AdRx - PD ROH -->\n                                           \n                <\/div>\n            <\/div>\n        <\/div>                   \n        <div class=\"panel\" style=\"height:600px !important;margin-top:10px\">\n            <div class=\"bannergroup\">\n              <div class=\"banneritem\"> \n                                                               \n                                 \n                <\/div>\n            <\/div>\n        <\/div>\n  <\/div>\n        \n                     \n  <\/div>  \n  <div id=\"footer\">\n    <div class=\"copyright\">\t\n\n<div class=\"custom\">\n\t<p>\n\tCopyright \u00a9 \t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<span class=\"cunudnxuzbfjfxemdzpj\"><\/span>\n\n\n2012 Healthdailyusa.com. 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