499 lines
42 KiB
Text
499 lines
42 KiB
Text
<!DOCTYPE html>
|
||
<html class="no-js" lang="en" dir="ltr" prefix="content: http://purl.org/rss/1.0/modules/content/ dc: http://purl.org/dc/terms/ foaf: http://xmlns.com/foaf/0.1/ rdfs: http://www.w3.org/2000/01/rdf-schema# sioc: http://rdfs.org/sioc/ns# sioct: http://rdfs.org/sioc/types# skos: http://www.w3.org/2004/02/skos/core# xsd: http://www.w3.org/2001/XMLSchema#">
|
||
<head>
|
||
<title>NIH launches trial for rare degenerative muscle disease treatment | National Institutes of Health (NIH)</title>
|
||
<meta charset="utf-8" />
|
||
<script type="text/javascript">
|
||
//<![CDATA[
|
||
var usasearch_config = { siteHandle:"nih" };
|
||
|
||
var script = document.createElement("script");
|
||
script.type = "text/javascript";
|
||
script.src = "//search.usa.gov/javascripts/remote.loader.js";
|
||
document.getElementsByTagName("head")[0].appendChild(script);
|
||
|
||
//]]>
|
||
</script><link rel="shortcut icon" href="https://www.nih.gov/favicon.ico" type="image/vnd.microsoft.icon" />
|
||
<meta name="google-site-verification" content="rjIoO4vW0AYgltSYvxM1L_CzGIkq28kHf9gHIASKx8s" />
|
||
<link rel="profile" href="http://www.w3.org/1999/xhtml/vocab" />
|
||
<meta http-equiv="cleartype" content="on" />
|
||
<link rel="apple-touch-icon" href="https://www.nih.gov/sites/all/themes/nih/apple-touch-icon.png" />
|
||
<meta name="description" content="Researchers have launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease, hereditary inclusion body myopathy (HIBM)." />
|
||
<meta name="keywords" content="NIH, degenerative muscle disease treatment, degenerative muscle disease" />
|
||
<meta name="robots" content="follow, index" />
|
||
<meta name="viewport" content="width=device-width, initial-scale=1.0" />
|
||
<meta name="generator" content="Drupal 7 (http://drupal.org)" />
|
||
<link rel="canonical" href="https://www.nih.gov/news-events/news-releases/nih-launches-trial-rare-degenerative-muscle-disease-treatment" />
|
||
<link rel="shortlink" href="https://www.nih.gov/node/25536" />
|
||
<meta http-equiv="content-language" content="EN" />
|
||
<meta property="og:site_name" content="National Institutes of Health (NIH)" />
|
||
<meta property="og:type" content="article" />
|
||
<meta property="og:title" content="NIH launches trial for rare degenerative muscle disease treatment" />
|
||
<meta property="og:url" content="https://www.nih.gov/news-events/news-releases/nih-launches-trial-rare-degenerative-muscle-disease-treatment" />
|
||
<meta property="og:description" content="Researchers have launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease, hereditary inclusion body myopathy (HIBM)." />
|
||
<meta property="og:updated_time" content="2016-04-06T16:29:35-04:00" />
|
||
<meta name="twitter:card" content="summary_large_image" />
|
||
<meta name="twitter:url" content="https://www.nih.gov/news-events/news-releases/nih-launches-trial-rare-degenerative-muscle-disease-treatment" />
|
||
<meta name="twitter:title" content="NIH launches trial for rare degenerative muscle disease treatment" />
|
||
<meta name="twitter:description" content="Researchers have launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease, hereditary inclusion body myopathy (HIBM)." />
|
||
<meta property="article:published_time" content="2015-09-30T19:17:47-04:00" />
|
||
<meta property="article:modified_time" content="2016-04-06T16:29:35-04:00" />
|
||
<meta name="published_date" content="2015-09-30T19:17:47-04:00" />
|
||
<meta name="last_revision" content="2016-04-06T16:29:35-04:00" />
|
||
<link type="text/css" rel="stylesheet" href="https://www.nih.gov/sites/default/files/css/css_PfvPCWanzn7FjDwZqwAHhPoOxYKwkQP-RZSK1pFFG0s.css" media="all" />
|
||
<link type="text/css" rel="stylesheet" href="https://www.nih.gov/sites/default/files/css/css__mIWP9gcStq74isjZVoAAsiih8VXwJoN75Nyw_BXrj0.css" media="screen" />
|
||
<style media="print">#sb-container{position:relative;}#sb-overlay{display:none;}#sb-wrapper{position:relative;top:0;left:0;}#sb-loading{display:none;}
|
||
</style>
|
||
<link type="text/css" rel="stylesheet" href="https://www.nih.gov/sites/default/files/css/css_jENQOQqZCRFGZK__DTefxYiXR2RUf03k1ugj3_Vg7vk.css" media="all" />
|
||
<link type="text/css" rel="stylesheet" href="//cdn.jsdelivr.net/qtip2/3.0.3/jquery.qtip.min.css" media="all" />
|
||
<link type="text/css" rel="stylesheet" href="https://www.nih.gov/sites/default/files/css/css_3yt-b0M1HNJsvKsXwYjh5TEtX2lJGAvJvtmVAfORYZs.css" media="all" />
|
||
<link type="text/css" rel="stylesheet" href="//fonts.googleapis.com/css?family=Droid+Sans:700,regular|Droid+Serif:italic,regular&subset=latin" media="all" />
|
||
<link type="text/css" rel="stylesheet" href="https://www.nih.gov/sites/default/files/css/css_E4VlyxyL3JMcT6dslWLGb0uQdoI2bh_Xj0N65V-bcWo.css" media="all" />
|
||
<link type="text/css" rel="stylesheet" href="https://www.nih.gov/sites/default/files/css/css_5rDMv37-JxnNA70cRr_1jlAjVs56DCb8EN4XB2QLl0c.css" media="all" />
|
||
|
||
<!--[if lte IE 8]>
|
||
<link type="text/css" rel="stylesheet" href="https://www.nih.gov/sites/default/files/css/css_aLJB_yHt22A_mQek4IRDNsur5tEkCpqLe72j_w6QHQ0.css" media="all" />
|
||
<![endif]-->
|
||
<link type="text/css" rel="stylesheet" href="https://www.nih.gov/sites/default/files/css/css_KviiBystpJcQq4MplcQZvCFxnu9V763f5bPq_AKh4xM.css" media="all" />
|
||
<script src="https://www.nih.gov/sites/default/files/js/js_mQAv54bz2JVXcwS5QWlz0fVY9Os67zeu9bgNqtlVLPY.js"></script>
|
||
<script src="//code.jquery.com/jquery-2.2.4.min.js"></script>
|
||
<script>window.jQuery || document.write("<script src='/sites/all/modules/contrib/jquery_update/replace/jquery/2.2/jquery.min.js'>\x3C/script>")</script>
|
||
<script>jQuery.migrateMute=true;jQuery.migrateTrace=false;</script>
|
||
<script src="//code.jquery.com/jquery-migrate-1.4.1.min.js"></script>
|
||
<script>window.jQuery && window.jQuery.migrateWarnings || document.write("<script src='/sites/all/modules/contrib/jquery_update/replace/jquery-migrate/1.4.1/jquery-migrate.min.js'>\x3C/script>")</script>
|
||
<script src="https://www.nih.gov/sites/default/files/js/js_9sGTPBGcOGZjLiSLGrYEnuUf8VdvLmL_DEZNNNbnHXk.js"></script>
|
||
<script src="https://www.nih.gov/sites/default/files/js/js_hNnF7a_sbk6nZ2-mOamHzalYND7HBHDLRInsaU93HDk.js"></script>
|
||
<script>document.createElement( "picture" );</script>
|
||
<script src="https://www.nih.gov/sites/default/files/js/js_gHk2gWJ_Qw_jU2qRiUmSl7d8oly1Cx7lQFrqcp3RXcI.js"></script>
|
||
<script src="//cdn.jsdelivr.net/qtip2/3.0.3/jquery.qtip.min.js"></script>
|
||
<script defer="defer" src="//script.crazyegg.com/pages/scripts/0011/2616.js"></script>
|
||
<script src="https://www.nih.gov/sites/default/files/js/js_3s0l7MQ3oYmGkNAypyzCJq3a0wT85XX_P1SLgnph5K4.js"></script>
|
||
<script>
|
||
Shadowbox.path = "/sites/all/libraries/shadowbox/";
|
||
</script>
|
||
<script>jQuery.extend(Drupal.settings, {"basePath":"\/","pathPrefix":"","setHasJsCookie":0,"instances":"{\u0022default\u0022:{\u0022content\u0022:{\u0022text\u0022:\u0022\u0022},\u0022style\u0022:{\u0022tip\u0022:false,\u0022classes\u0022:\u0022\u0022},\u0022position\u0022:{\u0022at\u0022:\u0022bottom right\u0022,\u0022adjust\u0022:{\u0022method\u0022:\u0022\u0022},\u0022my\u0022:\u0022top left\u0022,\u0022viewport\u0022:false},\u0022show\u0022:{\u0022event\u0022:\u0022mouseenter \u0022},\u0022hide\u0022:{\u0022event\u0022:\u0022mouseleave \u0022}}}","qtipDebug":"{\u0022leaveElement\u0022:0}","shadowbox":{"animate":true,"animateFade":true,"animSequence":"wh","auto_enable_all_images":1,"auto_gallery":1,"autoplayMovies":true,"continuous":false,"counterLimit":10,"counterType":"default","displayCounter":true,"displayNav":true,"enableKeys":true,"fadeDuration":0.34999999999999998,"handleOversize":"resize","handleUnsupported":"link","initialHeight":160,"initialWidth":320,"language":"en","modal":false,"overlayColor":"#000","overlayOpacity":0.80000000000000004,"resizeDuration":0.55000000000000004,"showMovieControls":true,"slideshowDelay":0,"viewportPadding":20,"useSizzle":false},"picture":{"mappings":[["Sidebar media mappings","sidebar_media_mappings"],["Floated media mappings","floated_media_mappings"],["Not Set","not_set"]],"label":"Image size (required)"},"extlink":{"extTarget":"_blank","extClass":"ext","extLabel":"(link is external)","extImgClass":0,"extIconPlacement":0,"extSubdomains":1,"extExclude":"(.\\.nih\\.gov.)|(.\\.cancer\\.gov.)|(.\\.genome\\.gov.)|(.\\.drugabuse\\.gov.)|(locatorplus\\.gov.)|(.\\.govdelivery\\.com.)|(.\\.hatchbuck\\.com.)|(nihseniorhealth\\.gov.)|(clinicaltrials\\.gov.)|(search\\.usa\\.gov\\\/search\\?affiliate\\=hip.)|(search\\.gov\\\/search\\?affiliate\\=hip.)|(drugabuse\\.gov.)|(medlineplus\\.gov.)|(collegedrinkingprevention\\.gov.)|(smokefree\\.gov.)|(arpa-h\\.gov.)|(nih\\.sharepoint\\.com)","extInclude":"(.\\.pagefreezer\\.com.)","extCssExclude":".media, .emergency-message, .slideshow-main, .bean-home-slideshow-2l-block","extCssExplicit":"","extAlert":"_blank","extAlertText":"You are leaving the NIH website.\r\n\r\nThis external link provides additional information that is consistent with the intended purpose of this site. NIH cannot attest to the accuracy of a non-federal site.\r\n\r\nLinking to a non-federal site does not constitute an endorsement by NIH or any of its employees of the sponsors or the information and products presented on the site. You will be subject to the destination site\u2019s privacy policy when you follow the link.","mailtoClass":"mailto","mailtoLabel":"(link sends e-mail)","extUseFontAwesome":0}});</script>
|
||
|
||
<script type="application/javascript" id="uws-snippet">
|
||
// Verint Unified WebSDK Integration Snippet v2.3
|
||
//
|
||
// Instructions: please embed this snippet directly into every page of your website template.
|
||
// For optimal performance, this should be embedded directly into the template, not referenced as
|
||
// an external file.
|
||
//
|
||
// DO NOT MODIFY BELOW THIS LINE *****************************************
|
||
(function () {
|
||
var cookies = document.cookie.split("; ").reduce(function (o, i) { var p = i.split("="); o[p[0]] = p[1]; return o; }, {});
|
||
var containerValue = cookies.uws_container_override && JSON.parse(decodeURIComponent(cookies.uws_container_override));
|
||
var config = {
|
||
moduleHost: "ucm-us.verint-cdn.com",
|
||
configHost: "ucm-us.verint-cdn.com",
|
||
siteKey: "nih-gov",
|
||
container: containerValue || "live",
|
||
loadTime: Date.now()
|
||
};
|
||
var readyCallbacks = []; window.uwsReady = function (callback) { readyCallbacks.push(callback); };
|
||
var sdkPath = "//" + [config.moduleHost, "files/sites", config.siteKey, config.container, "sdk.js"].join("/");
|
||
var script = document.createElement("script"); script.src = sdkPath; script.async = true; script.type = "module";
|
||
script.addEventListener("load", function () { window.unifiedSDK.start(config, readyCallbacks); });
|
||
document.head.appendChild(script);
|
||
})();
|
||
// DO NOT MODIFY ABOVE THIS LINE *****************************************
|
||
</script>
|
||
|
||
</head>
|
||
<body class="html not-front not-logged-in page-node page-node- page-node-25536 node-type-nih-news-release section-news-events">
|
||
<a href="#main-content" class="element-invisible element-focusable">Skip to main content</a>
|
||
<noscript aria-hidden="true"><iframe src="https://www.googletagmanager.com/ns.html?id=GTM-5CBCNW2" height="0" width="0" style="display:none;visibility:hidden"></iframe></noscript>
|
||
<div class="l-page has-one-sidebar has-sidebar-second">
|
||
|
||
<div class="l-page-content">
|
||
|
||
<header class="l-header" role="banner">
|
||
<div class="l-ribbon-wrapper">
|
||
<ul class="l-ribbon">
|
||
<li><span class="ribbon-hhs"></span><a href="https://www.hhs.gov">U.S. Department of Health & Human Services</a><span class="ribbon-right"></span></li>
|
||
</ul>
|
||
</div> <div class="l-banner-wrapper">
|
||
<div class="l-banner">
|
||
<div class="l-branding">
|
||
<a class="nih-logo icon-nih-logo" href="/" title="National Institutes of Health (NIH) - Turning Discovery into Health" rel="home"><img src="/sites/all/themes/nih/images/nih-logo-color.png" width="390" height="60" alt="National Institutes of Health (NIH) - Turning Discovery into Health" /></a>
|
||
</div>
|
||
<!-- DigitalGov Search -->
|
||
<form accept-charset="UTF-8" action="https://search.nih.gov/search" id="search" class="search-form site-search" method="get">
|
||
<div style="margin:0;padding:0;display:inline"><input name="utf8" type="hidden" value="✓" /></div>
|
||
<input id="affiliate" name="affiliate" type="hidden" value="nih" />
|
||
<label for="query" class="hide">Search the NIH Website</label>
|
||
<input autocomplete="on" class="usagov-search-autocomplete form-field form-field-site-search" id="query" name="query" type="text" placeholder="Search NIH" /><input type="submit" name="commit" value="Search" class="form-button form-button-site-search" />
|
||
</form>
|
||
<ul class="banner-utilities">
|
||
<li><a href="/virtual-tour">Virtual Tour</a></li>
|
||
<li><a href="https://ned.nih.gov/">Staff Directory</a></li>
|
||
<li><a href="https://salud.nih.gov">En Español</a></li>
|
||
</ul>
|
||
</div>
|
||
</div>
|
||
</header>
|
||
|
||
<div class="l-navigation-wrapper">
|
||
<!-- ****************** SITE MENU ****************** -->
|
||
<h2 class="element-invisible">Site Menu</h2>
|
||
<nav class="l-menu-wrapper">
|
||
<ul class="l-menu">
|
||
<!-- ********* HOME TAB (Mobile view only) ********* -->
|
||
<li class="tab-wrapper tab-wrapper-home">
|
||
<div class="tab home"><a href="/"><span>Home</span></a></div>
|
||
</li>
|
||
<!-- ********* HEALTH INFO TAB ********* -->
|
||
<li class="tab-wrapper tab-wrapper-healthinfo tab-wrapper-left">
|
||
<div class="tab"><a href="/health-information"><span>Health Information</span></a></div>
|
||
<div class="megamenu">
|
||
<div class="megamenu-col megamenu-leftcol">
|
||
<ul class="sub-menu">
|
||
<li><a href="https://www.hhs.gov/programs/social-services/health-care-facilities/index.html">Health Care Providers & Facilities</a></li>
|
||
<li><a href="/health-information/health-info-lines">Health Info Lines</a></li>
|
||
<li><a href="https://www.healthcare.gov">HealthCare.gov</a></li>
|
||
<li><a href="/research-training/science-education">Science Education Resources</a></li>
|
||
<li><a href="/health-information/nih-clinical-research-trials-you">NIH Clinical Research Trials and You</a></li>
|
||
<li><a href="/institutes-nih/nih-office-director/office-communications-public-liaison/clear-communication/talking-your-doctor">Talking to Your Doctor</a></li>
|
||
<!--<li><a href="/institutes-nih/nih-office-director/office-communications-public-liaison/clear-communication/clear-health-nih" title="">Clear Health A–Z</a></li>-->
|
||
</ul>
|
||
<p class="more-wrapper"><a class="button gold" href="/health-information" title="More Health Information">More »</a></p>
|
||
</div>
|
||
<div class="megamenu-col megamenu-rightcol">
|
||
<form id="hipsearch" accept-charset="UTF-8" action="https://search.nih.gov/search" method="get" class="search-form">
|
||
<p class="subhead"><label for="query2">Search Health Topics</label></p>
|
||
<div class="input-wrapper">
|
||
<div style="margin:0;padding:0;display:inline"><input name="utf8" type="hidden" value="✓" /></div>
|
||
<input id="affiliate" name="affiliate" type="hidden" value="hip" />
|
||
<input type="hidden" name="dc" value="940" />
|
||
<input autocomplete="on" maxlength="50" class="usagov-search-autocomplete form-field form-field-search" id="query2" name="query" type="text" />
|
||
<input name="commit" type="submit" value="Go" id="searchbutton" class="form-button" />
|
||
</div>
|
||
</form>
|
||
<p class="subhead">Quick Links</p>
|
||
<ul class="sub-menu">
|
||
<li><a class="minibadge minibadge-medlineplus" href="https://medlineplus.gov/"><span class="minibadge-text">MedlinePlus Health Info</span></a></li>
|
||
<li><a class="minibadge minibadge-newsinhealth" href="https://newsinhealth.nih.gov/"><span class="minibadge-text">NIH News in Health</span></a></li>
|
||
<li><a class="minibadge minibadge-wellnesstoolkits" href="/health-information/your-healthiest-self-wellness-toolkits"><span class="minibadge-text">Wellness Toolkits</span></a></li>
|
||
</ul>
|
||
</div>
|
||
</div>
|
||
</li>
|
||
<!-- ********* GRANTS TAB ********* -->
|
||
<li class="tab-wrapper tab-wrapper-grants tab-wrapper-left">
|
||
<div class="tab"><a href="/grants-funding"><span>Grants & Funding</span></a></div>
|
||
<div class="megamenu">
|
||
<div class="megamenu-col megamenu-leftcol">
|
||
<ul>
|
||
<li><a href="https://grants.nih.gov">Grants Home Page</a></li>
|
||
<li><a href="https://grants.nih.gov/funding/index.htm">Find Funding</a></li>
|
||
<li><a href="https://grants.nih.gov/grants/how-to-apply-application-guide/due-dates-and-submission-policies/due-dates.htm">Due Dates</a></li>
|
||
<li><a href="https://grants.nih.gov/grants/how-to-apply-application-guide.html">How to Apply</a></li>
|
||
<li><a href="https://grants.nih.gov/grants/about_grants.htm">About Grants</a></li>
|
||
<li><a href="https://grants.nih.gov/policy/index.htm">Policy & Compliance</a></li>
|
||
<li><a href="https://nexus.od.nih.gov/all/">Grants News/Blog</a></li>
|
||
<li><a href="https://grants.nih.gov/funding/contracts.htm">Contracts</a></li>
|
||
<li><a href="https://www.lrp.nih.gov/">Loan Repayment</a></li>
|
||
</ul>
|
||
<p class="more-wrapper"><a class="button gold" href="/grants-funding" title="More Grants & Funding Information">More »</a></p>
|
||
</div>
|
||
<div class="megamenu-col megamenu-rightcol">
|
||
<p class="subhead">Quick Links</p>
|
||
<ul>
|
||
<li><a class="minibadge minibadge-report" href="https://report.nih.gov/"><span class="minibadge-text">RePORT</span></a></li>
|
||
<li><a class="minibadge minibadge-era" href="https://commons.era.nih.gov/commons/"><span class="minibadge-text">eRA Commons</span></a></li>
|
||
<li><a class="minibadge minibadge-commonfund" href="https://commonfund.nih.gov/"><span class="minibadge-text">NIH Common Fund</span></a></li>
|
||
</ul>
|
||
</div>
|
||
</div>
|
||
</li>
|
||
<!-- ********* NEWS TAB ********* -->
|
||
<li class="tab-wrapper tab-wrapper-news tab-wrapper-left">
|
||
<div class="tab"><a href="/news-events"><span>News & Events</span></a></div>
|
||
<div class="megamenu">
|
||
<div class="megamenu-col megamenu-leftcol">
|
||
<ul>
|
||
<li><a href="/news-events/news-releases">News Releases</a></li>
|
||
<li><a href="/news-events/digital-media-kits">Digital Media Kits</a></li>
|
||
<li><a href="/news-events/media-resources">Media Resources</a></li>
|
||
<li><a href="/news-events/media-contacts">Media Contacts</a></li>
|
||
<li><a href="/news-events/images-b-roll">Images and B-roll</a></li>
|
||
<li><a href="/news-events/events">Events</a></li>
|
||
<li><a href="/news-events/social-media-outreach">Social Media</a></li>
|
||
</ul>
|
||
<p class="more-wrapper"><a class="button gold" href="/news-events" title="More News & Events">More »</a></p>
|
||
</div>
|
||
<div class="megamenu-col megamenu-rightcol">
|
||
<p class="subhead">Quick Links</p>
|
||
<ul>
|
||
<li><a class="minibadge minibadge-newsinhealth" href="https://newsinhealth.nih.gov/"><span class="minibadge-text">NIH News in Health</span></a></li>
|
||
<li><a class="minibadge minibadge-researchmatters" href="/news-events/nih-research-matters"><span class="minibadge-text">NIH Research Matters</span></a></li>
|
||
<li><a class="minibadge minibadge-nihrecord" href="https://nihrecord.nih.gov/"><span class="minibadge-text">NIH Record</span></a></li>
|
||
</ul>
|
||
</div>
|
||
</div>
|
||
</li>
|
||
<!-- ********* RESEARCH TAB ********* -->
|
||
<li class="tab-wrapper tab-wrapper-research tab-wrapper-right">
|
||
<div class="tab"><a href="/research-training"><span>Research & Training</span></a></div>
|
||
<div class="megamenu">
|
||
<div class="megamenu-col megamenu-leftcol">
|
||
<ul>
|
||
<li><a href="/research-training/medical-research-initiatives">Medical Research Initiatives</a></li>
|
||
<li><a href="/research-training/science-highlights">Science Highlights</a></li>
|
||
<li><a href="/research-training/science-education">Science Education</a></li>
|
||
<li><a href="/research-training/research-nih-labs-clinics">Research in NIH Labs & Clinics</a></li>
|
||
<li><a href="/research-training/training-opportunities">Training Opportunities</a></li>
|
||
<li><a href="/research-training/library-resources">Library Resources</a></li>
|
||
<li><a href="/research-training/research-resources">Research Resources</a></li>
|
||
<li><a href="/research-training/clinical-research-resources">Clinical Research Resources</a></li>
|
||
<li><a href="/research-training/safety-regulation-guidance">Safety, Regulation and Guidance</a></li>
|
||
</ul>
|
||
<p class="more-wrapper"><a class="button gold" href="/research-training" title="More Research & Training">More »</a></p>
|
||
</div>
|
||
<div class="megamenu-col megamenu-rightcol">
|
||
<p class="subhead">Quick Links</p>
|
||
<ul>
|
||
<li><a class="minibadge minibadge-pubmed" href="https://pubmed.ncbi.nlm.nih.gov"><span class="minibadge-text">PubMed</span></a></li>
|
||
<li><a class="minibadge minibadge-stemcell" href="https://stemcells.nih.gov"><span class="minibadge-text">Stem Cell Information</span></a></li>
|
||
<li><a class="minibadge minibadge-oppnet" href="https://oppnet.nih.gov"><span class="minibadge-text">OppNet</span></a></li>
|
||
<li><a class="minibadge minibadge-nidb" href="https://intramural.nih.gov"><span class="minibadge-text">NIDB</span></a></li>
|
||
<li><a class="minibadge minibadge-nihblueprint" href="https://neuroscienceblueprint.nih.gov"><span class="minibadge-text">NIH Blueprint for Neuroscience Research</span></a></li>
|
||
</ul>
|
||
</div>
|
||
</div>
|
||
</li>
|
||
<!-- ********* INSTITUTES TAB ********* -->
|
||
<li class="tab-wrapper tab-wrapper-institutes tab-wrapper-right">
|
||
<div class="tab"><a href="/institutes-nih"><span>Institutes at NIH</span></a></div>
|
||
<div class="megamenu">
|
||
<div class="megamenu-col megamenu-leftcol">
|
||
<ul>
|
||
<li><a href="/institutes-nih/list-institutes-centers">List of Institutes and Centers</a></li>
|
||
<li><a href="/institutes-nih/nih-office-director">NIH Office of the Director</a></li>
|
||
<li><a href="/institutes-nih/directors-nih-institutes-centers">Directors of NIH Institutes and Centers</a></li>
|
||
<li><a href="/institutes-nih/nih-institute-center-contact-information">NIH Institute and Center Contact Information</a></li>
|
||
</ul>
|
||
<p class="more-wrapper"><a class="button gold" href="/institutes-nih" title="More About the Institutes at NIH">More »</a></p>
|
||
</div>
|
||
<div class="megamenu-col megamenu-rightcol">
|
||
<p class="subhead">Quick Links</p>
|
||
<ul class="ic-acronymn-list">
|
||
<li><a href="https://www.cancer.gov" title="National Cancer Institute">NCI</a></li>
|
||
<li><a href="https://nei.nih.gov" title="National Eye Institute">NEI</a></li>
|
||
<li><a href="https://www.nhlbi.nih.gov" title="National Heart, Lung, and Blood Institute">NHLBI</a></li>
|
||
<li><a href="https://www.genome.gov" title="National Human Genome Research Institute">NHGRI</a></li>
|
||
<li><a href="https://www.nia.nih.gov" title="National Institute on Aging">NIA</a></li>
|
||
<li><a href="https://www.niaaa.nih.gov" title="National Institute on Alcohol Abuse and Alcoholism">NIAAA</a></li>
|
||
<li><a href="https://www.niaid.nih.gov" title="National Institute of Allergy and Infectious Diseases">NIAID</a></li>
|
||
<li><a href="https://www.niams.nih.gov" title="National Institute of Arthritis and Musculoskeletal and Skin Diseases">NIAMS</a></li>
|
||
<li><a href="https://www.nibib.nih.gov" title="National Institute of Biomedical Imaging and Bioengineering">NIBIB</a></li>
|
||
<li><a href="https://www.nichd.nih.gov" title="Eunice Kennedy Shriver National Institute of Child Health and Human Development">NICHD</a></li>
|
||
<li><a href="https://www.nidcd.nih.gov" title="National Institute on Deafness and Other Communication Disorders">NIDCD</a></li>
|
||
<li><a href="https://www.nidcr.nih.gov" title="National Institute of Dental and Craniofacial Research">NIDCR</a></li>
|
||
<li><a href="https://www.niddk.nih.gov" title="National Institute of Diabetes and Digestive and Kidney Diseases">NIDDK</a></li>
|
||
<li><a href="https://nida.nih.gov" title="National Institute on Drug Abuse">NIDA</a></li>
|
||
<li><a href="https://www.niehs.nih.gov" title="National Institute of Environmental Health Sciences">NIEHS</a></li>
|
||
<li><a href="https://www.nigms.nih.gov" title="National Institute of General Medical Sciences">NIGMS</a></li>
|
||
<li><a href="https://www.nimh.nih.gov" title="National Institute of Mental Health">NIMH</a></li>
|
||
<li><a href="https://www.nimhd.nih.gov" title="National Institute on Minority Health and Health Disparities">NIMHD</a></li>
|
||
<li><a href="https://www.ninds.nih.gov" title="National Institute of Neurological Disorders and Stroke">NINDS</a></li>
|
||
<li><a href="https://www.ninr.nih.gov" title="National Institute of Nursing Research">NINR</a></li>
|
||
<li><a href="https://www.nlm.nih.gov" title="National Library of Medicine">NLM</a></li>
|
||
<li><a href="https://www.cc.nih.gov" title="NIH Clinical Center">CC</a></li>
|
||
<li><a href="https://www.cit.nih.gov" title="Center for Information Technology">CIT</a></li>
|
||
<li><a href="https://www.csr.nih.gov" title="Center for Scientific Review">CSR</a></li>
|
||
<li><a href="https://www.fic.nih.gov" title="Fogarty International Center">FIC</a></li>
|
||
<li><a href="https://ncats.nih.gov" title="National Center for Advancing Translational Sciences">NCATS</a></li>
|
||
<li><a href="https://nccih.nih.gov" title="National Center for Complementary and Integrative Health">NCCIH</a></li>
|
||
</ul>
|
||
</div>
|
||
</div>
|
||
</li>
|
||
<!-- ********* ABOUT TAB ********* -->
|
||
<li class="tab-wrapper tab-wrapper-about tab-wrapper-right">
|
||
<div class="tab"><a href="/about-nih"><span>About NIH</span></a></div>
|
||
<div class="megamenu">
|
||
<div class="megamenu-col megamenu-leftcol">
|
||
<ul>
|
||
<li><a href="/about-nih/who-we-are">Who We Are</a></li>
|
||
<li><a href="/about-nih/what-we-do">What We Do</a></li>
|
||
<li><a href="https://hr.nih.gov/jobs">Jobs at NIH</a></li>
|
||
<li><a href="/about-nih/visitor-information">Visitor Information</a></li>
|
||
<li><a href="/about-nih/frequently-asked-questions">Frequently Asked Questions</a></li>
|
||
<li><a href="/about-nih/contact-us">Contact Us</a></li>
|
||
<!--<li><a href="/about-nih/explore-nih">Explore NIH</a></li>-->
|
||
</ul>
|
||
<p class="more-wrapper"><a class="button gold" href="/about-nih" title="More About NIH">More »</a></p>
|
||
</div>
|
||
<div class="megamenu-col megamenu-rightcol">
|
||
<p class="subhead">Quick Links</p>
|
||
<ul>
|
||
<li><a href="/about-nih/who-we-are/nih-director">The NIH Director</a></li>
|
||
<li><a href="/virtual-tour">Take the Virtual Tour</a></li>
|
||
<li><a href="/about-nih/what-we-do/nih-turning-discovery-into-health">NIH…Turning Discovery Into Health<sup>®</sup></a></li>
|
||
<li><a href="/about-nih/what-we-do/impact-nih-research">Impact of NIH Research</a></li>
|
||
<li><a href="/about-nih/what-we-do/science-health-public-trust">Science, Health, and Public Trust</a></li>
|
||
</ul>
|
||
</div>
|
||
</div>
|
||
</li>
|
||
</ul>
|
||
<div class="tab-bkgnd"></div><!-- For pixel rounding issue -->
|
||
</nav>
|
||
<!-- ****************** END SITE MENU ****************** -->
|
||
</div>
|
||
|
||
<div class="l-main-wrapper">
|
||
<div class="l-main" id="page-content">
|
||
<h2 class="element-invisible">You are here</h2><div class="breadcrumb"><a href="/">Home</a> » <a href="/news-events">News & Events</a> » <a href="/news-events/news-releases">News Releases</a></div> <div class="l-region l-region--section-header">
|
||
<div id="block-block-96" class="block block--block sectionheader block--block-96" id="block-block-96" class="block block--block sectionheader block--block-96">
|
||
|
||
|
||
<div class="content" class="block__content">
|
||
<h2><a href="/news-events/news-releases">News Releases</a></h2> </div>
|
||
</div>
|
||
</div>
|
||
<div class="l-content-wrapper">
|
||
<div class="l-content" role="main">
|
||
<a id="main-content"></a>
|
||
<div about="/news-events/news-releases/nih-launches-trial-rare-degenerative-muscle-disease-treatment" typeof="sioc:Item foaf:Document" class="ds-1col node node--nih-news-release news-release view-mode-full node--full node--nih-news-release--full clearfix">
|
||
|
||
|
||
<p class="publishdate"><span class="tag-term">News Release</span></p><p class="publishdate"><span property="dc:date" datatype="xsd:dateTime" content="2012-09-24T00:00:00-04:00" class="date-display-single">Monday, September 24, 2012</span></p><h1>NIH launches trial for rare degenerative muscle disease treatment</h1><p class="subtitle">
|
||
<p>Researchers have launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease, hereditary inclusion body myopathy (HIBM). National Institutes of Health scientists from the National Center for Advancing Translational Sciences (NCATS) and the National Human Genome Research Institute (NHGRI) will conduct the clinical trial at the NIH Clinical Center.</p>
|
||
|
||
<p>HIBM, also known as GNE myopathy, has no available therapy. Disease symptoms emerge in adulthood and slowly lead to progressive muscle weakness. Most patients develop symptoms in their early 20s and eventually require a wheelchair as their arm, hand and leg muscles weaken. Mutations in the GNE gene cause HIBM by producing low sialic acid levels in muscle proteins, which scientists think contributes to the symptoms of muscle weakness. Normally, GNE produces an enzyme that produces sialic acid, a sugar important to muscle development and kidney function.</p>
|
||
|
||
<p>“This study marks an important milestone toward developing a treatment for an underserved patient population, and we would not be this far along had it not been for the teamwork and dedication of the researchers working on this collaboration,” said Christopher P. Austin, M.D., the newly appointed NCATS director.</p>
|
||
|
||
<p>In 2007, Marjan Huizing, Ph.D., an associate investigator in NHGRI's Medical Genetics Branch, led a team of scientists in search of an HIBM treatment. They hypothesized that a compound called ManNAc, now called DEX-M74, might improve the low sialic acid levels that cause HIBM. DEX-M74 is a sugar that the body converts to sialic acid.</p>
|
||
|
||
<p>Huizing and colleagues conducted studies that showed the compound was effective in controlling sialic acid levels in a mouse model with a specific GNE mutation. The researchers published their findings in the June 2007 issue of the Journal of Clinical Investigation. Based on these results, they set out to evaluate the effects of DEX-M74 on progressive muscle weakness in HIBM patients. However, the project required additional funding for pre-clinical studies.</p>
|
||
|
||
<p>In 2009, NIH established its Therapeutics for Rare and Neglected Diseases (TRND) program, now part of NCATS, to facilitate the pre-clinical development of new drugs for these ailments. TRND scientists selected the development of DEX-M74 as a treatment for HIBM as one of its initial pilot projects. The collaboration includes TRND researchers, the laboratories of Marjan Huizing, Ph.D., and of William A. Gahl, M.D, Ph.D., principal investigator of NHGRI’s Medical Genetics Branch, and New Zealand Pharmaceuticals Limited (NZP). NZP is manufacturing DEX-M74, which was developed by Drs. Gahl and Huizing and licensed from the NIH by NZP.</p>
|
||
|
||
<p>"TRND infused life into the HIBM project by supporting pre-clinical studies for the investigational new drug application," said Dr. Gahl, who also serves as NHGRI clinical director. "The program provides the missing link in the evolution of drug treatments. It is a resource that has the potential to develop new therapeutics for rare diseases, often with applicability to common disorders."</p>
|
||
|
||
<p>During the HIBM project, the TRND program has supported toxicology studies to evaluate the safety of DEX-M74. Researchers also generated chemistry manufacturing and controls data, which relate to the formulation and manufacturing process of a drug. Based on the availability of these new data, the collaborators completed an IND application that the U.S. Food and Drug Administration recently allowed to go into effect.</p>
|
||
|
||
<p>"The TRND program was designed to provide the expertise and knowledge needed to advance potential treatments like DEX-M74 to human clinical trials," said John McKew, Ph.D., chief of the NCATS Therapeutic Development Branch and director of TRND. "The results of this project demonstrate what a translational program like TRND can accomplish through collaborations that bring experts together from basic research, pre-clinical drug development, and clinical medicine."</p>
|
||
|
||
<p>The HIBM Phase I clinical trial will test a single dose of DEX-M74 in a small group of patients with a focus on drug safety and how well patients tolerate the drug. Nuria Carrillo, M.D., TRND staff physician and principal investigator of the trial, plans to follow up the initial study with a Phase I/II trial in which patients will receive multiple doses of DEX-M74. Researchers will monitor patients for drug tolerance and indications of drug effectiveness. If DEX-M74 is safe in the Phase I/II trial, researchers will plan a Phase II study to determine the clinical effectiveness of the drug in HIBM patients.</p>
|
||
|
||
<p>"The NIH has achieved a significant milestone in the development of a potential treatment for HIBM, and we are excited about this research reaching the clinical trial stage," said NZP Chief Executive Officer Andy Lewis. "The pre-clinical data are very strong, and we are keen to see DEX-M74 progress through the clinical phases. Once we have proven human efficacy we plan to offer DEX-M74 to patients."</p>
|
||
|
||
<p>The HIBM clinical trial is the third TRND project to advance to human clinical trials. The two other clinical trials are evaluating treatments for sickle cell disease and chronic lymphocytic leukemia. Dr. Carrillo also is overseeing a natural history study of HIBM to collect health information from patients to understand how the disease develops. TRND has developed a portfolio of 14 projects, including HIBM, which focus on rare and neglected tropical diseases.</p>
|
||
|
||
<p>For more information about the HIBM Phase I clinical trial, please visit <a href="https://clinicaltrials.gov/ct2/show/NCT01634750?term=HIBM&rank=1">https://clinicaltrials.gov/ct2/show/NCT01634750?term=HIBM&rank=1</a>.</p>
|
||
|
||
<p>Information about the HIBM natural history study is available at <a href="https://clinicaltrials.gov/ct2/show/NCT01417533?term=TRND&rank=3">https://clinicaltrials.gov/ct2/show/NCT01417533?term=TRND&rank=3</a>.</p>
|
||
|
||
<p>To learn more about TRND clinical trials, please go to <a href="http://www.ncats.nih.gov/research/rare-diseases/trnd/studies/trnd-crs.html">http://www.ncats.nih.gov/research/rare-diseases/trnd/studies/trnd-crs.html</a>.</p>
|
||
|
||
<p>The National Center for Advancing Translational Sciences (NCATS) aims to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions. For more information about NCATS, visit <a href="http://www.ncats.nih.gov">http://www.ncats.nih.gov</a>.</p>
|
||
|
||
<p>NHGRI is one of the 27 institutes and centers at the National Institutes of Health. The NHGRI Division of Intramural Research develops and implements technology to understand, diagnose and treat genomic and genetic diseases. Additional information about NHGRI can be found at its website, <a href="http://www.genome.gov">http://www.genome.gov</a>.</p>
|
||
|
||
<p>About the NIH Clinical Center: The NIH Clinical Center is the 240-bed clinical research hospital for the National Institutes of Health. Through clinical research, clinician-investigators translate laboratory discoveries into better treatments, therapies, and interventions to improve the nation's health. For more information, visit <a href="http://clinicalcenter.nih.gov">http://clinicalcenter.nih.gov</a>.</p><p><strong>About the National Institutes of Health (NIH):</strong>
|
||
NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit <a href="/">www.nih.gov</a>.</p>
|
||
|
||
<p class="slogan"><em>NIH…Turning Discovery Into Health<sup>®</sup></em></p><p
|
||
class='hashtags'>###</p>
|
||
|
||
|
||
</div>
|
||
|
||
<div class="lastupdated">
|
||
</div> </div>
|
||
<aside class="l-region l-region--sidebar-second masonry-container">
|
||
<div id="block-ds-extras-nih-news-release-page-ds" class="block block--ds-extras block--ds-extras-nih-news-release-page-ds" id="block-ds-extras-nih-news-release-page-ds" class="block block--ds-extras block--ds-extras-nih-news-release-page-ds">
|
||
|
||
|
||
<div class="content" class="block__content">
|
||
<div class="box"><h3 class="label-above">Institute/Center</h3><p><a href="http://ncats.nih.gov/">National Center for Advancing Translational Sciences (NCATS)</a></p><p><a href="http://www.nhgri.nih.gov/">National Human Genome Research Institute (NHGRI)</a></p><p><a href="http://clinicalcenter.nih.gov/">NIH Clinical Center (CC)</a></p></div><div class="box"><h3 class="label-above">Contact</h3><div class="paragraph">
|
||
<div class="field--contact-name">
|
||
<a href="mailto:info@ncats.nih.gov">NCATS Office of Communications </a> </div>
|
||
|
||
<div class="field--contact-number">
|
||
301-435-0888 </div>
|
||
</div><div class="paragraph">
|
||
<div class="field--contact-name">
|
||
<a href="mailto:macdougallr@mail.nih.gov">Raymond MacDougall</a> </div>
|
||
|
||
<div class="field--contact-number">
|
||
301-402-0911 </div>
|
||
</div></div > </div>
|
||
</div>
|
||
<div id="block-bean-news-releases-connect-with-us" class="block block--bean box block--bean-news-releases-connect-with-us" id="block-bean-news-releases-connect-with-us" class="block block--bean box block--bean-news-releases-connect-with-us">
|
||
|
||
<h3 class="block__title">Connect with Us</h3>
|
||
|
||
<div class="content" class="block__content">
|
||
<div about="/block/news-releases---connect-with-us" class="ds-1col entity entity-bean bean-basic-block view-mode-default clearfix">
|
||
|
||
|
||
<ul class="icon-list">
|
||
<li><a class="icon-link email-monochrome" href="https://service.govdelivery.com/accounts/USNIH/subscriber/new?topic_id=USNIH_1" onclick="window.open(this.href,'popup','width=725,height=500,left=25,top=25,location=no,resizable=yes,scrollbars=yes,status=yes');return false;" target="_blank" title="Subscribe."><span class="icon"></span>Subscribe to news releases</a></li>
|
||
<li><a class="icon-link rss-monochrome" href="/news-releases/feed.xml"><span class="icon"></span>RSS Feed</a></li>
|
||
</ul></div>
|
||
|
||
</div>
|
||
</div>
|
||
</aside>
|
||
</div>
|
||
<div class="l-connect-wrapper">
|
||
<div class="l-connect">
|
||
<h2 class="element-invisible">Connect with Us</h2>
|
||
<ul>
|
||
<li><a class="contactus-monochrome" href="/about-nih/contact-us"><span class="icon"></span>Contact Us</a></li>
|
||
<li><a class="twitter-monochrome" href="https://twitter.com/NIH"><span class="icon"></span>X</a></li>
|
||
<li><a class="facebook-monochrome" href="https://www.facebook.com/nih.gov"><span class="icon"></span>Facebook</a></li>
|
||
<li><a class="instagram-monochrome" href="https://www.instagram.com/nihgov/"><span class="icon"></span>Instagram</a></li>
|
||
<li><a class="youtube-monochrome" href="https://www.youtube.com/user/nihod"><span class="icon"></span>YouTube</a></li>
|
||
<li><a class="flickr-monochrome" href="https://www.flickr.com/photos/nihgov/"><span class="icon"></span>Flickr</a></li>
|
||
<li><a class="social-monochrome" href="/news-events/social-media-outreach"><span class="icon"></span>More Social Media from NIH</a></li>
|
||
</ul>
|
||
</div>
|
||
</div>
|
||
</div>
|
||
</div>
|
||
|
||
<div class="l-footer-wrapper">
|
||
<footer class="l-footer" role="contentinfo">
|
||
<h2 class="element-invisible">Footer</h2>
|
||
<ul>
|
||
<li><a href="/">NIH Home</a></li>
|
||
<li><a href="/virtual-tour">Virtual Tour</a></li>
|
||
<li><a href="https://salud.nih.gov">En Español</a></li>
|
||
<li><a href="/about-nih/visitor-information">Visitor Information</a></li>
|
||
<li><a href="/about-nih/frequently-asked-questions">Frequently Asked Questions</a></li>
|
||
<li><a href="/privacy-policy">Privacy Policy</a></li>
|
||
<li><a href="/disclaimers">Disclaimers</a></li>
|
||
<li><a href="/accessibility">Accessibility</a></li>
|
||
<li><a href="/nih-website-archives">NIH Website Archives</a></li>
|
||
</ul>
|
||
<ul>
|
||
<li><a href="/nondiscrimination-notice">Nondiscrimination Notice</a></li>
|
||
<li><a href="/institutes-nih/nih-office-director/office-communications-public-liaison/freedom-information-act-office">Freedom of Information Act</a></li>
|
||
<li><a href="https://www.edi.nih.gov/no-fear-act">No Fear Act</a></li>
|
||
<li><a href="https://www.hhs.gov/vulnerability-disclosure-policy/index.html">HHS Vulnerability Disclosure</a></li>
|
||
<li><a href="https://oig.hhs.gov">Office of Inspector General</a></li>
|
||
<li><a class="lastTab" href="https://www.usa.gov/">USA.gov</a></li>
|
||
</ul>
|
||
<p>NIH…Turning Discovery Into Health<sup>®</sup></p>
|
||
<p>National Institutes of Health, 9000 Rockville Pike, Bethesda, Maryland 20892</p>
|
||
<p><a href="https://www.hhs.gov">U.S. Department of Health and Human Services</a></p>
|
||
</footer>
|
||
</div>
|
||
|
||
<div class="back-to-top"><a href="#"><span class="icon-arrow-up"></span><span class="text">Back to Top</span></a></div>
|
||
|
||
</div>
|
||
|
||
</div>
|
||
<script src="https://www.nih.gov/sites/default/files/js/js_7Ukqb3ierdBEL0eowfOKzTkNu-Le97OPm-UqTS5NENU.js"></script>
|
||
<script src="https://www.nih.gov/sites/default/files/js/js_E9yUYWK8EMydR0HMIp2PahVxPAClXyH0Cnh782A15UM.js"></script>
|
||
</body>
|
||
</html>
|
||
|