{"id":36621,"date":"2023-09-30T20:48:05","date_gmt":"2023-10-01T00:48:05","guid":{"rendered":"https:\/\/lakelandgazette.info\/news\/?p=36621"},"modified":"2023-09-30T20:48:08","modified_gmt":"2023-10-01T00:48:08","slug":"florida-nemours-childrens-hospital-treats-first-patient-with-gene-therapy","status":"publish","type":"post","link":"https:\/\/lakelandgazette.info\/news\/2023\/09\/30\/florida-nemours-childrens-hospital-treats-first-patient-with-gene-therapy\/","title":{"rendered":"Florida Nemours Children\u2019s Hospital treats first patient with gene therapy"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\">Nemours Children\u2019s Hospital, Florida, has been recognized as an official treatment site for gene therapy to treat a rare muscular disease.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">ELEVIDYS from the Sarepta Therapeutics is now used to treat Duchenne Muscular Dystrophy.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Sarepta said the therapy, called Elevidys, will cost $3.2 million per patient. It\u2019s designed to be taken just once.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Dr. Omar Abdul Hamid, a pediatric neurologist at Nemours Children\u2019s Hospital, treated the first patient in Florida with this disease.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DMD is caused by a genetic variant that affects keeping muscle cells intact. According to the hospital, Duchenne primarily affects males and about 250,000 to 300,000 people worldwide.<\/p>\n\n\n<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"480\" height=\"360\" sizes=\"auto, (max-width: 480px) 100vw, 480px\" src=\"https:\/\/lakelandgazette.info\/news\/wp-content\/uploads\/2023\/09\/image-7.jpg\" alt=\"\" class=\"wp-image-36622\" srcset=\"https:\/\/lakelandgazette.info\/news\/wp-content\/uploads\/2023\/09\/image-7.jpg 480w, https:\/\/lakelandgazette.info\/news\/wp-content\/uploads\/2023\/09\/image-7-300x225.jpg 300w\" \/><\/figure>\n<\/div>\n\n\n<p class=\"wp-block-paragraph\">Symptoms show in childhood with muscle weakness and can interfere with the ability to walk, leading to a lower life expectancy.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The U.S. Food and Drug Administration approved the treatment as the first gene therapy for DMD. There is no current cure for the disease, but Nemours\u2019 experts said they are hopeful it can halt disease progression.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cThis innovative, new gene therapy will treat the cause of the disease,\u201d Hamid said. \u201cAnd is another tool for us as providers to help treat these patients; we are happy to be able to offer it to our patients who are candidates.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Nemours Children\u2019s Hospital, Florida, has been recognized as an official treatment site for gene therapy to treat a rare muscular disease. ELEVIDYS from the Sarepta Therapeutics is now used to&hellip; <\/p>\n","protected":false},"author":2,"featured_media":36622,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"inline_featured_image":false,"_uf_show_specific_survey":0,"_uf_disable_surveys":false,"wpai_generated_summary":"","footnotes":"","jetpack_publicize_message":"{title}\n\n{excerpt}\n\n{url}","jetpack_publicize_feature_enabled":true,"jetpack_social_post_already_shared":true,"jetpack_social_options":{"image_generator_settings":{"template":"dois","default_image_id":71799,"font":"","enabled":false},"version":2},"_wpas_customize_per_network":false},"categories":[14],"tags":[6176],"class_list":["post-36621","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","tag-florida-nemours-childrens-hospital-treats-first-patient-with-gene-therapy"],"jetpack_publicize_connections":[],"_links":{"self":[{"href":"https:\/\/lakelandgazette.info\/news\/wp-json\/wp\/v2\/posts\/36621","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lakelandgazette.info\/news\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lakelandgazette.info\/news\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lakelandgazette.info\/news\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/lakelandgazette.info\/news\/wp-json\/wp\/v2\/comments?post=36621"}],"version-history":[{"count":0,"href":"https:\/\/lakelandgazette.info\/news\/wp-json\/wp\/v2\/posts\/36621\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/lakelandgazette.info\/news\/wp-json\/wp\/v2\/media\/36622"}],"wp:attachment":[{"href":"https:\/\/lakelandgazette.info\/news\/wp-json\/wp\/v2\/media?parent=36621"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lakelandgazette.info\/news\/wp-json\/wp\/v2\/categories?post=36621"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lakelandgazette.info\/news\/wp-json\/wp\/v2\/tags?post=36621"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}