{"id":4088,"date":"2016-10-24T12:54:48","date_gmt":"2016-10-24T12:54:48","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=4088"},"modified":"2022-05-03T18:25:18","modified_gmt":"2022-05-03T18:25:18","slug":"utrophin","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/utrophin\/","title":{"rendered":"Utrophin production might be a good option for people with DMD"},"content":{"rendered":"<p>Utrophin could potentially replace dystrophin. In people with Duchenne muscular dystrophy (DMD), dystrophin does not function properly. Numerous media outlets have recently reported many exciting developments in the search for new treatments for Duchenne muscular dystrophy (DMD). Science, medicine, and technology are uniting to create significant results. This article is the first in a new series to shed light on promising, experimental treatment options. This first article will feature utrophin production.<\/p>\n<h3>What is utrophin?<\/h3>\n<p>The human body naturally produces utrophin, a protein,\u00a0<span class=\"s1\">when a muscle is first forming or when a muscle is repairing<\/span>.\u00a0<span class=\"s1\">As a muscle matures<\/span>, dystrophin replaces utrophin. However, in people with Duchenne muscular dystrophy (DMD), dystrophin does not function properly.<\/p>\n<p>Utrophin is functionally and structurally similar to dystrophin. Preclinical trials that have stimulated\u00a0<span class=\"s1\">sustained\u00a0<\/span>utrophin production have shown that it could potentially replace dystrophin in people with Duchenne muscular dystrophy (DMD). The replacement of dysfunctional dystrophin with functional utrophin might have a highly positive impact on muscle performance.<\/p>\n<p>Summit Therapeutics believes that utrophin may slow or even stop the progression of DMD.<\/p>\n<p>More information: <a href=\"http:\/\/www.parentprojectmd.org\/site\/PageServer?pagename=Advance_research_strategies_utrophin\">PPMD<\/a>\u00a0 &#8211; \u00a0<a href=\"https:\/\/en.wikipedia.org\/wiki\/Utrophin\">Wikipedia<\/a>\u00a0 &#8211; <a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pubmed\/8866746\">NCBI<\/a><\/p>\n<h3>What is ezutromid?<\/h3>\n<p>Ezutromid is an utrophin modulator.<\/p>\n<p class=\"p1\"><span class=\"s1\">It is an orally administered, small molecule, experimental drug,\u00a0<\/span><span class=\"s1\">in development<\/span><span class=\"s1\"> by Summit Therapeutics.<\/span><\/p>\n<p><a href=\"http:\/\/www.summitplc.com\/programmes\/duchenne-muscular-dystrophy\/\">More information<\/a><\/p>\n<h3>How does it work?<\/h3>\n<p>Ezutromid stimulates the body\u00a0<span class=\"s1\">to sustain\u00a0<\/span>production of utrophin.<\/p>\n<p>This experimental therapy has the potential to work in people with all genetic profiles of Duchenne muscular dystrophy (DMD).\u00a0Given that it doesn\u2019t depend on a specific genetic profile, this treatment may be suitable for 100% of people with DMD.<\/p>\n<h3>Clinical Trial<\/h3>\n<p class=\"p1\"><span class=\"s1\">Summit Therapeutics\u00a0just announced that the first patients have been enrolled into trial sites in the US into PhaseOut DMD. Summit Therapeutics\u00a0will be making updates about additional US sites and contact details\u00a0to the\u00a0<a href=\"http:\/\/clinicaltrials.gov\/\"><span class=\"s2\">clinicaltrials.gov<\/span><\/a> record (<a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02858362\"><span class=\"s2\">https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02858362<\/span><\/a>). As a reminder, PhaseOut DMD is a 48-week, open-label (meaning all participants receive ezutromid) Phase 2 trial,\u00a0<\/span><span class=\"s1\">is ongoing in the UK and the US. Enrolment of approximately 40 patients continues, and they\u00a0expect to complete trial enrolment in the second quarter of 2017.<\/span><\/p>\n<p>For more information about PhaseOut DMD clinical trial of ezutromid:\u00a0<a href=\"http:\/\/www.utrophintrials.com\">Utrophin Trials<\/a> \u00a0 &#8211; \u00a0<a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02858362\">Clinical trials<\/a><\/p>\n<h3>What\u2019s the regulatory status\u00a0of ezutromid?<\/h3>\n<p class=\"p1\"><span class=\"s1\">Ezutromid is an experimental drug candidate in Phase 2 clinical trial<\/span><span class=\"s2\">.\u00a0<\/span>The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted Orphan Drug designation to ezutromid. Orphan drugs receive a number of benefits, including additional regulatory support and a period of market exclusivity after marketing approval. In addition, the FDA has granted ezutromid both Fast Track and Rare Pediatric Disease designations.<\/p>\n<p class=\"p1\">Recently, Summit Therapeutics Plc\u00a0applied to the MHRA and FDA regulatory authorities to proceed with the planned extension of PhaseOut DMD for patients currently enrolled in the trial. The extension phase is expected to last until ezutromid either receives marketing approval in relevant countries or its development is discontinued.<\/p>\n<p class=\"p1\"><span class=\"s1\">In addition to PhaseOut DMD, they\u00a0plan to conduct a randomised, placebo-controlled trial designed with the potential to support accelerated and conditional approvals for ezutromid in the US and Europe. It is anticipated that this trial would start after positive interim data from PhaseOut DMD.<\/span><\/p>\n<p class=\"p1\">Summit to Extend Ongoing\u00a0<span id=\"lblTitle\">PhaseOut DMD Clinical Trial of Ezutromid in Patients with DMD: <a href=\"http:\/\/otp.investis.com\/clients\/uk\/summit_corporation_plc\/rns\/regulatory-story.aspx?cid=1575&amp;newsid=857360\" target=\"_blank\" rel=\"noopener noreferrer\">Press Release\u00a0<\/a><\/span><\/p>\n<h3>How is ezutromid administered?<\/h3>\n<p>Ezutromid is\u00a0<span class=\"s1\">given orally, as a liquid.<\/span><\/p>\n<p><a href=\"http:\/\/musculardystrophynews.com\/2016\/08\/24\/summit-positive-phase-1-data-on-ezutromid-new-formulation-for-dmd\/\">Muscular Dystrophy News<\/a><\/p>\n<h3>A word from the manufacturer<\/h3>\n<p>\u201cThe Rare Pediatric Disease designation builds upon the Fast Track and Orphan Drug designations, which the FDA has already awarded to ezutromid, recognizing a significant unmet medical need in the treatment of DMD,\u201d\u00a0says Glyn Edwards, Chief Executive Officer of Summit.<\/p>\n<p>\u201cWe plan to leverage these regulatory advantages in the continued clinical development of ezutromid, which is currently in Phase 2 clinical trial called PhaseOut DMD, to bring ezutromid to patients in need as quickly as possible.\u201d<\/p>\n<p>For more information about ezutromid click <a href=\"http:\/\/finance.yahoo.com\/news\/summit-receives-rare-pediatric-disease-11000661.html\">here<\/a><\/p>\n<p>&nbsp;<\/p>\n<p>To learn more about new treatments you can subscribe\u00a0to our newsletter <a href=\"\/?page_id=3379\">here<\/a><\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Utrophin could potentially replace dystrophin. In people with Duchenne muscular dystrophy (DMD), dystrophin does not function properly. Numerous media outlets have recently reported many exciting developments in the search for new treatments for Duchenne muscular dystrophy (DMD). Science, medicine, and technology are uniting to create significant results. This article is the first in a new [&hellip;]<\/p>\n","protected":false},"author":2,"featured_media":4825,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[12,17,18],"tags":[],"class_list":["post-4088","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","category-research","category-treatments"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.6 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Utrophin could potentially replace dystrophin, a good option for DMD.<\/title>\n<meta name=\"description\" content=\"Utrophine could potentially replace dystrophin. In people with Duchenne muscular dystrophy (DMD), dystrophin does not fonction properly.\" \/>\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\/utrophin\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Utrophin could potentially replace dystrophin, a good option for DMD.\" \/>\n<meta property=\"og:description\" content=\"Utrophine could potentially replace dystrophin. 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