{"id":166493,"date":"2023-06-27T06:24:05","date_gmt":"2023-06-27T11:24:05","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2023\/06\/first-gene-therapy-to-treat-duchenne-muscular-dystrophy-is-approved"},"modified":"2023-06-27T06:24:05","modified_gmt":"2023-06-27T11:24:05","slug":"first-gene-therapy-to-treat-duchenne-muscular-dystrophy-is-approved","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2023\/06\/first-gene-therapy-to-treat-duchenne-muscular-dystrophy-is-approved","title":{"rendered":"First Gene Therapy to Treat Duchenne Muscular Dystrophy is Approved"},"content":{"rendered":"<p><\/p>\n<p><iframe style=\"display: block; margin: 0 auto; width: 100%; aspect-ratio: 4\/3; object-fit: contain;\" src=\"https:\/\/www.youtube.com\/embed\/YGHaW112RXI?feature=oembed\" frameborder=\"0\" allow=\"accelerometer; autoplay; encrypted-media; gyroscope;\n   picture-in-picture\" allowfullscreen><\/iframe><\/p>\n<p>The first gene therapy that can treat Duchenne muscular dystrophy (DMD) has been approved by the U.S. Food and Drug Administration; it will be marketed as Elevidys (delandistrogene moxeparvovec-rokl) by Sarepta Therapeutics Inc. Children aged four to five with the disorder and confirmed gene mutations will be eligible to receive the one-time treatment if insurers approve, as the cost is $3.2 million per patient.<\/p>\n<p>DMD is caused by mutations in a gene called dystrophin, which results in a serious lack of functional dystrophin protein. The gene therapy works by sending a gene that can produce a shortened version of the dystrophin protein to patients; the company has termed it Elevidys micro-dystrophin.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>The first gene therapy that can treat Duchenne muscular dystrophy (DMD) has been approved by the U.S. Food and Drug Administration; it will be marketed as Elevidys (delandistrogene moxeparvovec-rokl) by Sarepta Therapeutics Inc. Children aged four to five with the disorder and confirmed gene mutations will be eligible to receive the one-time treatment if insurers [\u2026]<\/p>\n","protected":false},"author":662,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,412],"tags":[],"class_list":["post-166493","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-genetics"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/166493","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/662"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=166493"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/166493\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=166493"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=166493"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=166493"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}