{"id":7458,"date":"2018-12-20T13:16:35","date_gmt":"2018-12-20T13:16:35","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=7458"},"modified":"2022-05-03T18:13:56","modified_gmt":"2022-05-03T18:13:56","slug":"news-about-golodirsen-skipping-exon-53","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/news-about-golodirsen-skipping-exon-53\/","title":{"rendered":"News about Golodirsen, Skipping Exon 53"},"content":{"rendered":"<h4>Sarepta Therapeutics completes submission of New Drug Application seeking approval of golodirsen in patients with DMD amenable to skipping exon 53<\/h4>\n<p>Press Release <a href=\"http:\/\/investorrelations.sarepta.com\/news-releases\/news-release-details\/sarepta-therapeutics-completes-submission-new-drug-application?fbclid=IwAR3O8SIAIB2HUWb4syvB6dZzUyoIhRRckAzwh35dphNdmn2pPR94fxEKztE\" rel=\"noreferrer noopener\">here<\/a><\/p>\n<p>Golodirsen has been studied for the treatment of exon 53 amenable patients, approximately eight percent of patients with DMD. Submission represents ongoing advancement of the company\u2019s proprietary PMO RNA-based platform.<\/p>\n<p>Sarepta Therapeutics, Inc.\u00a0announced today that it had completed the submission of its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053), a phosphorodiamidate morpholino oligomer engineered to treat those patients with Duchenne muscular dystrophy who have genetic mutations subject to skipping exon 53 of the Duchenne gene.<\/p>\n<p>If the golodirsen NDA is filed and granted accelerated approval, the company\u2019s ESSENCE study (4045-301) could serve as a post-marketing confirmatory study. ESSENCE, which is underway, is a global, randomized double-blind, placebo-controlled study assessing the safety and efficacy of golodirsen and casimersen, their exon 45 skipping therapy.<\/p>\n<p class=\"has-text-color has-background has-small-font-size has-very-dark-gray-color has-very-light-gray-background-color\"><strong>&#8220;We are grateful for the patients and clinicians who have participated in the study with an aim to advance treatment for all patients with Duchenne,\u201d <br \/>&#8220;Sarepta is committed to developing therapies to benefit the greatest possible percentage of patients affected by Duchenne. Our proprietary PMO technology remains central to our commitment to patients with Duchenne. Combined, EXONDYS 51\u00ae (eteplirsen), golodirsen, and casimersen have the potential to treat nearly 30 percent of patients with Duchenne.&#8221; Said DOUGLAS S. INGRAM, President &amp; CEO<\/strong><\/p>\n<h3><strong>About Golodirsen<\/strong><\/h3>\n<p>Golodirsen exon-skipping technology to skip exon 53 of the DMD gene. Exon skipping is intended to allow for the production of an internally truncated but functional dystrophin protein.<\/p>\n<p>Golodirsen uses Sarepta\u2019s proprietary phosphorodiamidate morpholino oligomer (PMO)<strong>*<\/strong>\u00a0chemistry and exon-skipping technology to skip exon\u00a053 of the\u00a0<em>DMD<\/em>\u00a0gene. Golodirsen is designed to bind to exon\u00a053 of dystrophin pre-mRNA, resulting in exclusion, or \u201cskipping,\u201d of this exon during mRNA processing in patients with genetic mutations that are amenable to exon\u00a053 skipping. Exon skipping is intended to allow for the production of an internally truncated but functional dystrophin protein.<\/p>\n<p>Golodirsen is one of the investigational candidates currently being evaluated in the ESSENCE study, a global, randomized, double-blind, placebo-controlled study evaluating efficacy and safety in patients amenable to skipping exons\u00a045 or 53.<\/p>\n<p><em><strong>*<\/strong>a phosphorodiamidate Morpholino oligomer\u00a0(PMO), is a type of\u00a0oligomer\u00a0molecule\u00a0(colloquially, an\u00a0oligo) used in\u00a0molecular biology\u00a0to modify\u00a0gene expression.\u00a0<\/em><\/p>\n<h3>More about dystrophin<\/h3>\n<p>Dystrophin is a protein found in muscle cells that, while present in extremely small amounts (about 0.002 percent of total muscle protein), is crucial in strengthening and protecting muscle fibers. A devastating and incurable muscle-wasting disease, DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Sarepta Therapeutics completes submission of New Drug Application seeking approval of golodirsen in patients with DMD amenable to skipping exon 53 Press Release here Golodirsen has been studied for the treatment of exon 53 amenable patients, approximately eight percent of patients with DMD. Submission represents ongoing advancement of the company\u2019s proprietary PMO RNA-based platform. Sarepta [&hellip;]<\/p>\n","protected":false},"author":2,"featured_media":7478,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[12,17],"tags":[],"class_list":["post-7458","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","category-research"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>news-about-golodirsen-skipping-exon-53<\/title>\n<meta name=\"description\" content=\"Sarepta Therapeutics completes submission of New Drug Application seeking approval of golodirsen in patients with DMD amenable to skipping exon 53\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/laforcedmd.com\/en\/news-about-golodirsen-skipping-exon-53\/\" \/>\n<meta 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