{"id":1694,"date":"2018-01-08T19:07:09","date_gmt":"2018-01-08T19:07:09","guid":{"rendered":"http:\/\/naiaonline.org\/blog\/?p=1694"},"modified":"2018-01-08T19:24:07","modified_gmt":"2018-01-08T19:24:07","slug":"myotubular-myopathy-clinical-trials-showing-early-success-in-children","status":"publish","type":"post","link":"https:\/\/naiaonline.org\/blog\/animal-research\/myotubular-myopathy-clinical-trials-showing-early-success-in-children\/","title":{"rendered":"Myotubular Myopathy Clinical Trials Showing Early Success in Children"},"content":{"rendered":"<p>Early <a href=\"http:\/\/www.kiro7.com\/video?videoId=677626174&amp;videoVersion=1.0\" target=\"_blank\" rel=\"noopener\">human clinical trials treating myotubular myopathy are showing great promise<\/a>, and have even exceeded expectations. This heartbreaking disease causes muscle weakness, difficulty breathing and eating, and abnormal bone development. Its victims, almost exclusively male, rarely live past early childhood.<\/p>\n<p><iframe loading=\"lazy\" src=\"http:\/\/w3.cdn.anvato.net\/player\/prod\/v3\/anvload.html?key=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%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%3D%3D\" width=\"400\" height=\"225\" frameborder=\"0\" scrolling=\"no\" allowfullscreen=\"allowfullscreen\"><\/iframe><br \/>\nOver the last two years, we had the privilege of seeing presentations by Drs. David Mack and Casey Childers, who had success in combating this disease in dogs with gene therapy (work made possible in large part by the efforts\u00a0<a href=\"https:\/\/www.joshuafrase.org\/about\/leadership.php\" target=\"_blank\" rel=\"noopener\">Alison\u00a0Rockett Frase<\/a>). The results were dramatic, and we all hoped for similar results in the human children who are affected, too. We are so incredibly proud of their work, and optimistic for future success!<\/p>\n<p><a href=\"http:\/\/www.naiaonline.org\/get-involved\/naia-videos\/david-mack-naia-conference-2016\" target=\"_blank\" rel=\"noopener\">Click here to see Dr. Mack&#8217;s presentation at the 2016 NAIA conference<\/a>.<\/p>\n<p>&nbsp;<\/p>\n<p><img decoding=\"async\" loading=\"lazy\" class=\"aligncenter size-medium wp-image-1697\" src=\"http:\/\/naiaonline.org\/blog\/wp-content\/uploads\/2018\/01\/NAIALogoTransparent-300x135.png\" alt=\"\" width=\"300\" height=\"135\" srcset=\"https:\/\/naiaonline.org\/blog\/wp-content\/uploads\/2018\/01\/NAIALogoTransparent-300x135.png 300w, https:\/\/naiaonline.org\/blog\/wp-content\/uploads\/2018\/01\/NAIALogoTransparent-768x347.png 768w, https:\/\/naiaonline.org\/blog\/wp-content\/uploads\/2018\/01\/NAIALogoTransparent-1024x462.png 1024w\" sizes=\"(max-width: 300px) 100vw, 300px\" \/><\/p>\n<!-- AddThis Advanced Settings generic via filter on the_content --><!-- AddThis Share Buttons generic via filter on the_content -->","protected":false},"excerpt":{"rendered":"<p>Over the last two years, we had the privilege of seeing presentations by Drs. David Mack and Casey Childers, who had success in combating this disease in dogs with gene therapy.<!-- AddThis Advanced Settings generic via filter on wp_trim_excerpt --><!-- AddThis Share Buttons generic via filter on wp_trim_excerpt --><\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":[],"categories":[15],"tags":[],"aioseo_notices":[],"_links":{"self":[{"href":"https:\/\/naiaonline.org\/blog\/wp-json\/wp\/v2\/posts\/1694"}],"collection":[{"href":"https:\/\/naiaonline.org\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/naiaonline.org\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/naiaonline.org\/blog\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/naiaonline.org\/blog\/wp-json\/wp\/v2\/comments?post=1694"}],"version-history":[{"count":6,"href":"https:\/\/naiaonline.org\/blog\/wp-json\/wp\/v2\/posts\/1694\/revisions"}],"predecessor-version":[{"id":1701,"href":"https:\/\/naiaonline.org\/blog\/wp-json\/wp\/v2\/posts\/1694\/revisions\/1701"}],"wp:attachment":[{"href":"https:\/\/naiaonline.org\/blog\/wp-json\/wp\/v2\/media?parent=1694"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/naiaonline.org\/blog\/wp-json\/wp\/v2\/categories?post=1694"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/naiaonline.org\/blog\/wp-json\/wp\/v2\/tags?post=1694"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}