CINXE.COM
Resources for Rare Disease Patients and Advocates | National Center for Advancing Translational Sciences
<!DOCTYPE html> <html lang="en" dir="ltr" prefix="og: https://ogp.me/ns#"> <head> <meta charset="utf-8" /> <meta name="description" content="Discover a wealth of resources tailored to support individuals affected by rare diseases and their advocates. Access information on patient registries, clinical trials, patient advocacy organizations, and other valuable tools aimed at assisting those navigating the challenges of rare diseases." /> <meta name="keywords" content="rare diseases; patient registry; clinical trial; patient advocacy" /> <link rel="canonical" href="http://ncats.nih.gov/resources/for-rare-disease-patients-and-advocates" /> <meta name="google" content="PROqkJCbtNawPO29xzL4ENjcb0_EckySw96i97QXx8s" /> <meta name="google-site-verification" content="PROqkJCbtNawPO29xzL4ENjcb0_EckySw96i97QXx8s" /> <meta name="Generator" content="Drupal 10 (https://www.drupal.org)" /> <meta name="MobileOptimized" content="width" /> <meta name="HandheldFriendly" content="true" /> <meta name="viewport" content="width=device-width, initial-scale=1.0" /> <link rel="icon" href="/themes/custom/d9_subtheme/assets/images/favicon.ico" type="image/vnd.microsoft.icon" /> <title>Resources for Rare Disease Patients and Advocates | National Center for Advancing Translational Sciences</title> <link rel="stylesheet" media="all" href="/libraries/drupal-superfish/css/superfish.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/ajax-progress.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/align.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/autocomplete-loading.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/fieldgroup.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/container-inline.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/clearfix.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/details.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/hidden.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/item-list.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/js.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/nowrap.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/position-container.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/progress.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/reset-appearance.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/resize.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/sticky-header.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/system-status-counter.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/system-status-report-counters.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/system-status-report-general-info.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/tabledrag.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/tablesort.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/system/css/components/tree-child.module.css?srmo5o" /> <link rel="stylesheet" media="all" href="/modules/contrib/extlink/extlink.css?srmo5o" /> <link rel="stylesheet" media="all" href="/core/modules/layout_discovery/layouts/onecol/onecol.css?srmo5o" /> <link rel="stylesheet" media="all" href="/modules/contrib/paragraphs/css/paragraphs.unpublished.css?srmo5o" /> <link rel="stylesheet" media="all" href="https://cdn.jsdelivr.net/npm/@uswds/uswds@3.3.0/dist/css/uswds.min.css" /> <link rel="stylesheet" media="all" href="https://cdn.jsdelivr.net/npm/bootstrap@5.0.2/dist/css/bootstrap.min.css" /> <link rel="stylesheet" media="all" href="https://cdn.jsdelivr.net/npm/bootstrap-icons@1.11.1/font/bootstrap-icons.css" /> <link rel="stylesheet" media="all" href="/themes/custom/d9_subtheme/css/styles.css?srmo5o" /> <script src="https://dap.digitalgov.gov/Universal-Federated-Analytics-Min.js?agency=HHS&subagency=NIH" id="_fed_an_ua_tag" async type="text/javascript"></script> </head> <body class="path-node page-node-type-landing-page-new-"> <a href="#main-content" class="usa-sr-only focusable"> Skip to main content </a> <noscript><iframe src="https://www.googletagmanager.com/ns.html?id=GTM-55CGV66V" height="0" width="0" style="display:none;visibility:hidden"></iframe></noscript> <div class="dialog-off-canvas-main-canvas" data-off-canvas-main-canvas> <section class="navigation-top"> <div class="container"> <div class="row"> <div class="col-12"> <div class="link-small d-flex"> <a href="http://www.hhs.gov/" title="Go to the hhs.gov" class="" target="_blank"> <img src="/sites/default/files/2023-10/hhs-white.png" alt="HHS Logo"> <span class="d-xl-inline d-lg-inline d-none small">U.S. Department of Health & Human Services</span> </a> <a href="http://www.nih.gov/" title="National Institutes of Health | Homepage" class="" target="_blank"> <img src="/sites/default/files/2023-10/nih-logo.png" alt="NIH Logo"> <span class="d-xl-inline d-lg-inline d-none small">National Institutes of Health</span> </a> </div> </div> </div> </div> </section> <div class="usa-overlay"></div> <header class="usa-header usa-header--extended usa-header--extended-megamenu" id="header" role="banner"> <div class="region region-header grid-container"> <div class="grid-row grid-gap-1"><div class="desktop:grid-col-3 header-first"> <div class="usa-logo site-logo" id="logo"> <em class="usa-logo__text"> <a class="logo-img" href="/" accesskey="1" title="Home" aria-label="Home"> <img src="/themes/custom/d9_subtheme/logo.png" alt="Home" /> </a> </em> </div> <div class="search-icon d-md-none"><a class="toggle-search" href="#"><i class="bi bi-search"></i></a></div> </div><div class="desktop:grid-col-7"> <div id="block-navigation" class="block block-superfish block-superfishnavigation"> <ul id="superfish-navigation" class="menu sf-menu sf-navigation sf-horizontal sf-style-none"> <li id="navigation-menu-link-contentaed842ec-f581-4cea-b0b0-4a85c50def86" class="sf-depth-1 menuparent"><a href="/research" class="sf-depth-1 menuparent">Research</a><ul><li id="navigation-menu-link-content5a3b6aac-7ae9-45d7-929f-fbf776db20f0" class="sf-depth-2 sf-no-children"><a href="/research/overview" class="sf-depth-2">Research Overview</a></li><li id="navigation-menu-link-contentd0507d8b-7b88-453c-908a-2b7e961c0479" class="sf-depth-2 sf-no-children"><a href="/research/our-impact" class="sf-depth-2">Our Impact</a></li><li id="navigation-menu-link-contented530d1e-2ec3-46db-a529-9029be3f3fb4" class="sf-depth-2 sf-no-children"><a href="/research/research-activities" class="sf-depth-2">Research Activities</a></li><li id="navigation-menu-link-content2dac8545-38f2-47c0-8993-ea0cae65e123" class="sf-depth-2 sf-no-children"><a href="/research/research-resources" class="sf-depth-2">Research Resources</a></li><li id="navigation-menu-link-content311ec98e-6232-4e22-9966-4a9ac796870a" class="sf-depth-2 sf-no-children"><a href="/research/training-education" class="sf-depth-2">Training & Education</a></li><li id="navigation-menu-link-content52b1ba41-94ea-4d84-9db7-7482b8f78f16" class="sf-depth-2 sf-no-children"><a href="/research/alliances" class="sf-depth-2">Alliances</a></li><li id="navigation-menu-link-contenta185af75-90a5-4cc4-813f-ef66f35e21ea" class="sf-depth-2 sf-no-children"><a href="/research/collaborate-with-ncats" class="sf-depth-2">Collaborate With NCATS</a></li></ul></li><li id="navigation-menu-link-content84a3cc1f-203d-4de6-ab26-d80c256b96dc" class="sf-depth-1 menuparent"><a href="/funding" class="sf-depth-1 menuparent">Funding</a><ul><li id="navigation-menu-link-content0a09bed2-3d99-4faf-b3c5-fc9a4878629a" class="sf-depth-2 sf-no-children"><a href="/funding/overview" class="sf-depth-2">Funding Overview</a></li><li id="navigation-menu-link-contentd85a1f38-7893-4e4a-8514-5b9e767983e2" class="sf-depth-2 sf-no-children"><a href="/funding/funding-opportunities" class="sf-depth-2">Open Funding Opportunities</a></li><li id="navigation-menu-link-content81e84c00-ef73-4aff-a15f-7728cc398801" class="sf-depth-2 sf-no-children"><a href="/funding/small-business-programs" class="sf-depth-2">Small Business Programs</a></li><li id="navigation-menu-link-content631a166b-650b-450f-97b4-9014d14935e9" class="sf-depth-2 sf-no-children"><a href="/funding/funding-policy-operating-guidelines" class="sf-depth-2">Funding Policy & Operating Guidelines</a></li><li id="navigation-menu-link-content5d56ebe7-b3e8-452a-bd30-df92b0deabef" class="sf-depth-2 sf-no-children"><a href="/funding/grant-information" class="sf-depth-2">Grant Information</a></li><li id="navigation-menu-link-content4429fdc9-e911-4022-b3dc-3f6a4980b9ff" class="sf-depth-2 sf-no-children"><a href="/funding/challenges" class="sf-depth-2">Challenges and Prize Competitions</a></li><li id="navigation-menu-link-content943fe682-94ff-4fcf-a6b0-32096d2b2d0e" class="sf-depth-2 sf-no-children"><a href="/funding/scientific-review" class="sf-depth-2">Scientific Review</a></li><li id="navigation-menu-link-content10496705-320e-4437-b2ba-ea2830b293d1" class="sf-depth-2 sf-no-children"><a href="/funding/conference-grant-information" class="sf-depth-2">Conference Grant Information</a></li></ul></li><li id="navigation-menu-link-contentea91f2ef-1b8d-4b1c-83e7-5830769dd505" class="active-trail sf-depth-1 menuparent"><a href="/resources" class="sf-depth-1 menuparent">Resources For You</a><ul><li id="navigation-menu-link-content567e64e3-685d-41bc-8bf4-130c7689d6a2" class="sf-depth-2 sf-no-children"><a href="/resources/overview" title="Resources for You" class="sf-depth-2">Resources for You</a></li><li id="navigation-menu-link-content786ce1f5-ff7a-463a-bb05-c01882062426" class="sf-depth-2 sf-no-children"><a href="/resources/for-researchers" title="Resources for Researchers" class="sf-depth-2">Resources for Researchers</a></li><li id="navigation-menu-link-contente737ec67-ecd0-4f18-915d-b61660cb524f" class="sf-depth-2 sf-no-children"><a href="/resources/for-trainees" title="Resources for Trainees" class="sf-depth-2">Resources for Trainees</a></li><li id="navigation-menu-link-content8812fdb6-0fb7-4b27-8401-2fc5b3f41ff1" class="active-trail sf-depth-2 sf-no-children"><a href="/resources/for-rare-disease-patients-and-advocates" title="Resources for Rare Disease Patients and Advocates" class="is-active sf-depth-2">Resources for Rare Disease Patients and Advocates</a></li></ul></li><li id="navigation-menu-link-contenta8527040-df23-4117-a5a7-566bd83e2c7f" class="sf-depth-1 menuparent"><a href="/news-events" class="sf-depth-1 menuparent">News & Events</a><ul><li id="navigation-menu-link-contentf6836a81-a42a-4528-b569-18939d8451da" class="sf-depth-2 sf-no-children"><a href="/news-events/overview" class="sf-depth-2">News & Events Overview</a></li><li id="navigation-menu-link-content9f44edc6-e5b7-4a92-bcb3-c0a8777461f4" class="sf-depth-2 sf-no-children"><a href="/news-events/news" class="sf-depth-2">News</a></li><li id="navigation-menu-link-content402cff9f-12b7-459b-9f0f-56606930399c" class="sf-depth-2 sf-no-children"><a href="/news-events/newsletter" class="sf-depth-2">Newsletters</a></li><li id="navigation-menu-link-content6cd76cbe-053e-4e3b-8961-c9f5def57ba6" class="sf-depth-2 sf-no-children"><a href="/news-events/events" class="sf-depth-2">Events</a></li><li id="navigation-menu-link-content0a211675-2fb5-45cc-b443-c7946e7d9120" class="sf-depth-2 sf-no-children"><a href="/news-events/science-publications" class="sf-depth-2">Scientific Publications</a></li><li id="navigation-menu-link-contente6d4ed4f-826f-41b4-a9e2-728c2fb4ca62" class="sf-depth-2 sf-no-children"><a href="/news-events/multimedia" class="sf-depth-2">Multimedia</a></li></ul></li><li id="navigation-menu-link-contentf1eb1fa8-5813-433c-9bcc-dc56f2e258f3" class="sf-depth-1 menuparent"><a href="/about" class="sf-depth-1 menuparent">About NCATS</a><ul><li id="navigation-menu-link-contentada9a195-ae5a-446a-9014-c1232164e6db" class="sf-depth-2 sf-no-children"><a href="/about/ncats-overview" class="sf-depth-2">NCATS Overview</a></li><li id="navigation-menu-link-contentd5e0369a-aded-4348-84f7-463c64498447" class="sf-depth-2 sf-no-children"><a href="/about/about-translational-science" class="sf-depth-2">About Translational Science</a></li><li id="navigation-menu-link-contente298b7b2-724e-408b-9bb9-afcaac57a751" class="sf-depth-2 sf-no-children"><a href="/about/advisory-committees" class="sf-depth-2">Advisory Committees</a></li><li id="navigation-menu-link-contentbc9c91b7-8d3d-4d2c-af65-c25ef894d269" class="sf-depth-2 sf-no-children"><a href="/about/budget" class="sf-depth-2">Budget</a></li><li id="navigation-menu-link-contenta4ed4439-05db-4a41-9357-731c2ff74543" class="sf-depth-2 sf-no-children"><a href="/about/director" class="sf-depth-2">Director's Corner</a></li><li id="navigation-menu-link-contentb50f96af-3723-4634-8566-4e60c2639491" class="sf-depth-2 sf-no-children"><a href="/about/divisions-and-offices" class="sf-depth-2">Divisions & Offices</a></li><li id="navigation-menu-link-content41d11ad4-c576-46be-a326-1f8cf5059354" class="sf-depth-2 sf-no-children"><a href="/about/our-staff" class="sf-depth-2">Our Staff</a></li></ul></li> </ul> </div> </div> <div class="desktop:grid-col-2 search-block"> <form class="usa-search usa-search--small" action="/search/node" method="get" id="search-block-form" accept-charset="UTF-8"> <div role="search"> <input class="usa-input" id="extended-search-field-small" type="search" name="keys" /> <button class="usa-button" type="submit"> <img src="/themes/custom/d9_subtheme/node_modules/@uswds/uswds/dist/img/usa-icons-bg/search--white.svg" class="usa-search__submit-icon" alt="Search"> </button> </div> </form> </div></div> </div> <nav class="usa-nav" role="navigation"> <div class="usa-nav__inner"> <button type="button" class="usa-nav__close"> <img src="/themes/custom/d9_subtheme/node_modules/@uswds/uswds/dist/img/usa-icons/close.svg" alt="close"/> </button> </div> </nav> </header> <main class="main-content usa-layout-docs usa-section " role="main" id="main-content"> <div class="grid-container-"> <div class="grid-row"> </div> <div class=""> </div> <div class="grid-row "> </div> <div class="grid-row grid-gap"> <div class="usa-layout-docs__main desktop:grid-col-fill"> <div class=""> <div data-drupal-messages-fallback class="hidden"></div> <div id="block-d9-subtheme-content" class="block block-system block-system-main-block"> <article data-history-node-id="27385"> <div> <div class="container layout layout--onecol"> <div class="layout__region layout__region--content"> <div class="block block-system block-system-breadcrumb-block"> <nav class="usa-breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="usa-sr-only">Breadcrumb</h2> <ol class="usa-breadcrumb__list"> <li class="usa-breadcrumb__list-item"> <a href="/" class="usa-breadcrumb__link"><span>Home</span></a> </li> <li class="usa-breadcrumb__list-item"> <a href="/resources/overview" class="usa-breadcrumb__link"><span>Resources For You</span></a> </li> <li class="usa-breadcrumb__list-item"> <span>Resources For Rare Disease Patients and Advocates</span> </li> </ol> </nav> </div> <div class="block block-layout-builder block-field-blocknodelanding-page-new-field-metatag"> </div> </div> </div> <div class="layout layout--onecol"> <div class="layout__region layout__region--content"> <div class="block block-layout-builder block-field-blocknodelanding-page-new-field-landing-page-components"> <section class="main-hero" style="background-image: linear-gradient(to left, rgba(255,255,255, 0.1), rgba(0, 0, 0, 0.99)), url('/sites/default/files/2023-09/RD%20Banner_revised_1440x350%20-%20Banner%20Images.png');"> <div class="container"> <div class="row"> <div class="col-12"> <div class="hero-content"> <h1>Resources for Rare Disease Patients and Advocates</h1> <p class="text-white"> We support and provide materials and information about rare diseases. Explore our rare diseases resources to find ways to get involved and learn about the latest research. </p> </div> </div> </div> </div> </section> <style type="text/css"> article {position: relative;} .block-system-breadcrumb-block{ position:absolute; top:0; color:#fff; } .block-system-breadcrumb-block .usa-breadcrumb__list, .block-system-breadcrumb-block .usa-breadcrumb__list a { color:#fff; } .block-system-breadcrumb-block .usa-breadcrumb__list .usa-breadcrumb__list-item:after { background-color:#fff !important; } </style> <div class="container subnav"> <nav role="navigation" aria-labelledby="block-navigation-menu" id="block-navigation"> <h2 class="usa-sr-only" id="block-navigation-menu">Navigation</h2> <ul class="menu menu--navigation nav"> <li class="first"> <a href="/resources/overview" title="Resources for You" class="first" data-drupal-link-system-path="node/27381">Resources for You</a> </li> <li> <a href="/resources/for-researchers" title="Resources for Researchers" data-drupal-link-system-path="node/27384">Resources for Researchers</a> </li> <li> <a href="/resources/for-trainees" title="Resources for Trainees" data-drupal-link-system-path="node/27382">Resources for Trainees</a> </li> <li class="active active-trail last"> <a href="/resources/for-rare-disease-patients-and-advocates" title="Resources for Rare Disease Patients and Advocates" class="active active-trail last active-trail is-active" data-drupal-link-system-path="node/27385">Resources for Rare Disease Patients and Advocates</a> </li> </ul> </nav> </div> <section> <div class="container news-content Advancing"> <div class="paragraph paragraph--type--body-content paragraph--view-mode--default"> <style> h2 { margin: 0 0 1rem 0; } hr.section-divider { margin: 1.5rem 0 1.5rem 0; clear: both; } .mt-4 { margin-top: 0!important; } </style> </div> </div> </section> <section> <div class="container news-content Advancing"> <div class="row desc-links"> <div class="col-12"> <h2>Rare Diseases Information</h2> <div class="researcher-text description"> <p>Speeding the development of treatments requires new solutions and working together. We are applying translational science approaches to address the public health challenge of rare diseases.</p> </div> <div class="desc-link"><a href="/research/our-impact/our-impact-rare-diseases">Explore Rare Diseases Information</a></div> </div> </div> </div> </section> <section> <div class="container news-content Advancing"> <div class="row vertical-line two-col"> <div class="col-xl-6 col-wrap"> <div class="pe-lg-4 p-0 col"> <h2>Genetic and Rare Diseases (GARD) Information Center</h2> <img src="/sites/default/files/2023-10/NCATS-2023_Recreate-GARD-Website-Image_620x275_V1.jpg" class="w-100 mt-2"> <div class="description pt-4"> <p>The GARD Information Center provides current and reliable health information on a wide range of genetic and rare diseases.</p> </div> <div class="text-center button-wrap"> <button class="buttons btn-1 mt-4"><a href="/research/research-resources/gard">Learn More About GARD</a></button> </div> </div> </div> <div class="col-xl-6 col-wrap"> <div class="ps-lg-4 p-0 col"> <h2>NCATS Toolkit for Patient-Focused Therapy Development</h2> <img src="/sites/default/files/2023-09/Toolkit_620x275.png" class="w-100 mt-2"> <div class="description pt-4"> <p>Our Toolkit for Patient-Focused Therapy Development is a collection of online resources to assist patient groups in the research and development of new treatments.</p> </div> <div class="text-center button-wrap"> <button class="buttons btn-1 mt-4"><a href="/research/research-resources/ncats-toolkit">Access the NCATS Toolkit</a></button> </div> </div> </div> </div> <hr class="section-divider"> </div> </section> <section> <div class="container news-content Advancing"> <h2 class="section-heading">Facts and Stats</h2> <div class="row resourse-row icon-cards"> <div class="col-xl-6 col-lg-6 mb-4 "> <div class="card icon-card card-link"> <div class="d-flex align-items-center"> <article class="media media--type-image media--view-mode-default"> <img loading="lazy" src="/sites/default/files/styles/large/public/2023-09/Fact_sheets_and_infographics-iconv2.png?itok=4fHvYIdN" width="57" height="67" alt="Icon of a magnifying glass over a person" /> </article> </div> <div class="ps-3"> <a class="cardlink stretched-link" href="/news-events/infographics-and-fact-sheets"> <h6>Fact Sheets and Infographics</h6></a> <p class="m-0"> Get the facts about rare diseases. </p> </div> </div> </div> <div class="col-xl-6 col-lg-6 mb-4 "> <div class="card icon-card card-link"> <div class="d-flex align-items-center"> <article class="media media--type-image media--view-mode-default"> <img loading="lazy" src="/sites/default/files/styles/large/public/2023-09/Directors_messages-icon_0.png?itok=7Wyf8PD-" width="60" height="55" alt="Icon of two people sitting at a table with a cog wheel floating above" /> </article> </div> <div class="ps-3"> <a class="cardlink stretched-link" href="/about/director/messages?category%5B0%5D=rare-diseases-research"> <h6>Director鈥檚 Messages</h6></a> <p class="m-0"> See messages from the NCATS director about rare diseases. </p> </div> </div> </div> </div> <hr class="section-divider"> </div> </section> <section> <div class="container news-content Advancing"> <h2 class="section-heading">Explore Community Resources</h2> <div class="description"> <p><span style="white-space:pre-wrap;">We provide a range of free materials and resources to help patients, caregivers, patient support organizations, health care providers and scientists learn about rare diseases and advance research in this area.</span></p> </div> <div class="row mb-4 three-pic-cards"> <div class="col-xl-4 ps-xl-3 mb-4"> <div class="card border-0 p-0 radius-10 h-100"> <div class="row p-20 g-4 align-items-stretch"> <div class="col-xl-12 col-lg-6"> <img src="/sites/default/files/2023-09/RaDaR_%20746x370.png" class="img-fluid"> </div> <div class="col-xl-12 col-lg-6"> <div class="card-body px-0 pt-0"> <h3 class="">Rare Diseases Registry Program (RaDaR)</h3> <p class="py-xl-0 py-lg-4 py-md-4 py-sm-4 py-2"> <p><span style="white-space:pre-wrap;">This resource provides best practices for setting up and maintaining patient registries for research purposes.</span></p> </p> </div> <div class="card-footer bg-white border-0 p-0"> <a href="/research/research-activities/radar">Learn About RaDaR</a> </div> </div> </div> </div> </div> <div class="col-xl-4 ps-xl-3 mb-4"> <div class="card border-0 p-0 radius-10 h-100"> <div class="row p-20 g-4 align-items-stretch"> <div class="col-xl-12 col-lg-6"> <img src="/sites/default/files/2023-09/RDD%20at%20NIH_blur%20names_746x370.png" class="img-fluid"> </div> <div class="col-xl-12 col-lg-6"> <div class="card-body px-0 pt-0"> <h3 class="">Rare Disease Day at NIH</h3> <p class="py-xl-0 py-lg-4 py-md-4 py-sm-4 py-2"> <p><span style="white-space:pre-wrap;">Rare Disease Day at NIH aims to raise awareness about rare diseases, the people they affect, and NIH collaborations that address scientific challenges and advance research for new treatments.</span></p> </p> </div> <div class="card-footer bg-white border-0 p-0"> <a href="/news-events/events/rdd">Learn About Rare Disease Day</a> </div> </div> </div> </div> </div> <div class="col-xl-4 ps-xl-3 mb-4"> <div class="card border-0 p-0 radius-10 h-100"> <div class="row p-20 g-4 align-items-stretch"> <div class="col-xl-12 col-lg-6"> <img src="/sites/default/files/2023-09/RD%20News_746x370.png" class="img-fluid"> </div> <div class="col-xl-12 col-lg-6"> <div class="card-body px-0 pt-0"> <h3 class="">Rare Disease News</h3> <p class="py-xl-0 py-lg-4 py-md-4 py-sm-4 py-2"> <p><span style="white-space:pre-wrap;">Our latest news stories showcase the rare diseases research we conduct and support.</span></p> </p> </div> <div class="card-footer bg-white border-0 p-0"> <a href="/news-events/news?keyword=rare">Read Our Stories</a> </div> </div> </div> </div> </div> </div> <hr class="section-divider"> </div> </section> <section> <div class="container news-content Advancing"> <h2 class="section-heading">Learn About Rare Diseases Research at NCATS</h2> <div class="description"> <p><span style="white-space:pre-wrap;">We support research programs and engage in activities to address the public health challenges of rare diseases.</span></p> </div> <div class="row mb-4 three-pic-cards"> <div class="col-xl-4 ps-xl-3 mb-4"> <div class="card border-0 p-0 radius-10 h-100"> <div class="row p-20 g-4 align-items-stretch"> <div class="col-xl-12 col-lg-6"> <img src="/sites/default/files/2023-09/Gene%20Therapy_746x370.png" class="img-fluid"> </div> <div class="col-xl-12 col-lg-6"> <div class="card-body px-0 pt-0"> <h3 class="">Gene-Targeted Therapy Programs</h3> <p class="py-xl-0 py-lg-4 py-md-4 py-sm-4 py-2"> <p><span style="white-space:pre-wrap;">We support and participate in multiple programs that speed the development of gene-targeted therapies for many diseases at a time.</span></p> </p> </div> <div class="card-footer bg-white border-0 p-0"> <a href="/research/research-activities?keyword=BGTC%20PaVe-GT%20Somatic">Learn About Gene-Targeted Therapy Programs</a> </div> </div> </div> </div> </div> <div class="col-xl-4 ps-xl-3 mb-4"> <div class="card border-0 p-0 radius-10 h-100"> <div class="row p-20 g-4 align-items-stretch"> <div class="col-xl-12 col-lg-6"> <img src="/sites/default/files/2023-09/TRND_746x370.png" class="img-fluid"> </div> <div class="col-xl-12 col-lg-6"> <div class="card-body px-0 pt-0"> <h3 class="">Therapeutics for Rare and Neglected Diseases (TRND)</h3> <p class="py-xl-0 py-lg-4 py-md-4 py-sm-4 py-2"> <p><span style="white-space:pre-wrap;">Through this in-house program, we support preclinical development of therapeutic options for diseases with high unmet medical needs.</span></p> </p> </div> <div class="card-footer bg-white border-0 p-0"> <a href="/research/research-activities/trnd">Learn More About TRND</a> </div> </div> </div> </div> </div> <div class="col-xl-4 ps-xl-3 mb-4"> <div class="card border-0 p-0 radius-10 h-100"> <div class="row p-20 g-4 align-items-stretch"> <div class="col-xl-12 col-lg-6"> <img src="/sites/default/files/2024-05/RDCRN_746x370_0.png" class="img-fluid"> </div> <div class="col-xl-12 col-lg-6"> <div class="card-body px-0 pt-0"> <h3 class="">Rare Diseases Clinical Research Network (RDCRN)</h3> <p class="py-xl-0 py-lg-4 py-md-4 py-sm-4 py-2"> <p><span style="white-space:pre-wrap;">We oversee this NIH-wide program that supports medical research on over 200 rare diseases through clinical studies, collaborations, study enrollment and data sharing.</span> </p> </p> </div> <div class="card-footer bg-white border-0 p-0"> <a href="/research/research-activities/rdcrn">Learn About RDCRN</a> </div> </div> </div> </div> </div> </div> </div> </section> </div> <div class="block block-layout-builder block-extra-field-blocknodelanding-page-new-content-moderation-control"> </div> </div> </div> </div> </article> </div> <div id="block-footercss" class="block block-block-content block-block-content2db33373-6fb7-49c5-be2f-83266eddea39"> <style type="text/css"> ul#superfish-navigation li { line-height:1.25; } .region-header .block-superfish ul.sf-menu li a {padding-right:40px !important;} @media (min-width: 1200px) { ul.sf-menu.sf-navigation li.menuparent ul {width:280px !important;} } .main-content article .menu--navigation { display:flex; flex-wrap: nowrap; justify-content: space-between; align-items: stretch; } .main-content article .menu--navigation li { flex: 1 1 0; width: 0; align-items: center; display: flex; justify-content: center; } .main-content article .menu--navigation li .dropdown-toggle { white-space: unset; } .image-box img, div.col-md-4.col-lg-2 img { height: auto; } .caption-left-md { float: left; width: 50%; margin: 0 1% 2% 0; border: 1px solid #ddd; } .caption-left-md img { width: 100%!important; height: auto; } div.block.block-layout-builder.block-field-blocknodestafffield-divisions-office { display: none } @media (max-width: 992px) { .main-content article .menu--navigation { flex-direction:column; } .main-content article .menu--navigation li { display:inline-block; margin:0; padding:.25rem; width: auto; text-align:left; } .main-content article .menu--navigation li:hover, .main-content article .menu--navigation li.active { border-bottom: 4px solid transparent !important; } .main-content article .menu--navigation li:hover a, .main-content article .menu--navigation li.active a { border-bottom:4px solid #00626f !important; padding-bottom:4px; } } p.last-update-container { font-size: 15px; font-style: italic; } </style> </div> </div> </div> </div> </div> </main> <div> <div id="block-d9-subtheme-lastupdateblock" class="block block-ncats-common block-last-update-block"> <div class="container"><p class="last-update-container">Last updated on October 11, 2024</p></div> </div> </div> <footer class="pt-4 footer mt-4"> <div class="container"> <div class="row"> <div class="col-12 py-4"> <img src="/sites/default/files/2023-10/ncats-logo-footer.png" class="img-fluid" alt="NCATS"> </div> </div> <div class="row"> <div class="container"> <div class="row justify-content-between"> <div class="col-xl-4"> <div class="pt-4"> <address> 9609 Medical Center Drive<br> Rockville, MD 20850-9793<br> 301-594-8966<br> <a href="mailto:ncatsinfo@nih.gov">ncatsinfo@nih.gov</a> </address> </div> <div class="text-white social-links mt-4"> <h5>Connect with us</h5> <ul id="social-media-icons" class="list-unstyled d-flex mb-4"> <li><a href="http://www.facebook.com/ncats.nih.gov" title="Visit us on Facebook" target="_blank"><i class="bi bi-facebook"></i></a></li> <li><a href="https://www.linkedin.com/company/nih-ncats" title="Visit us on LinkedIn" target="_blank"><i class="bi bi-linkedin"></i></a></li> <li><a href="http://www.twitter.com/ncats_nih_gov" title="Visit us on X" target="_blank"><i class="bi bi-twitter-x"></i></a></li> <li><a href="http://www.youtube.com/user/ncatsmedia" title="View our videos on YouTube" target="_blank"><i class="bi bi-youtube"></i></a></li> <li><a href="https://www.flickr.com/photos/64860478@N05/" target="_blank" title="View our photos on Flickr"><img src="/sites/default/files/2023-10/flickr.png" alt="View our photos on Flickr" style="height:24px;"></a></li> </ul> <p class="small">For <a href="http://edi.nih.gov/consulting/language-access-program/about" target="_blank">language access assistance</a>, contact the <a href="mailto:ncatsinfo@nih.gov">NCATS Public Information Officer</a></p> </div> </div> <div class="col-xl-3 text-white mt-5 d-lg-none d-sm-none d-none d-xl-block"> <div id="mc_embed_shell"> <div id="mc_embed_signup"> <form action="https://nih.us5.list-manage.com/subscribe/post?u=e06bd55ccbb41f1a362306dff&id=d32fa404b4&f_id=0087b1edf0" method="post" id="mc-embedded-subscribe-form" name="mc-embedded-subscribe-form" class="validate" target="_blank"> <h5>Subscribe to NCATS News</h5> <p>If you would like to stay up to date with the latest information from us, please sign up for NCATS News.</p> <div id="mce-responses" class="clear"> <div class="response" id="mce-error-response" style="display: none;"></div> <div class="response" id="mce-success-response" style="display: none;"></div> </div> <div class="input-group mb-3"> <input type="email" name="EMAIL" class="required email form-control" id="mce-EMAIL" required="" value="" placeholder="Enter Email" aria-label="Recipient's username" aria-describedby="button-addon2" tabindex="-1"> <div aria-hidden="true" style="position: absolute; left: -5000px;"><input type="text" name="b_e06bd55ccbb41f1a362306dff_d32fa404b4" tabindex="-1" value=""></div> <button type="submit" name="subscribe" id="mc-embedded-subscribe" class="button btn" value="Subscribe">Submit</button> </div> </form> </div> <script type="text/javascript" src="//s3.amazonaws.com/downloads.mailchimp.com/js/mc-validate.js"></script><script type="text/javascript">(function($) {window.fnames = new Array(); window.ftypes = new Array();fnames[0]='EMAIL';ftypes[0]='email';fnames[1]='FNAME';ftypes[1]='text';fnames[2]='LNAME';ftypes[2]='text';fnames[3]='SIGNUP';ftypes[3]='text';}(jQuery));var $mcj = jQuery.noConflict(true);</script></div> </div> </div> </div> </div> </div> <!-- footer menu section --> <section class="footer-menu py-3 mb-xl-0 mb-lg-0 mb-sm-5 mt-4"> <div class="container"> <div class="row"> <div class="col-12"> <ul class="list-unstyled d-xl-flex d-lg-flex d-sm-block d-block justify-content-center mb-0"> <li><a href="https://ncats.nih.gov/">NCATS Home</a></li> <li><a href="/sitemap">Site Map</a></li> <li><a href="/privacy">Privacy Notice</a></li> <li><a href="/comments">Comment Policy</a></li> <li><a href="/accessibility">Accessibility</a></li> <li><a href="/foia">FOIA</a></li> <li><a href="http://oig.hhs.gov/" target="_blank">OIG</a></li> <li class="border-0"><a href="https://www.hhs.gov/vulnerability-disclosure-policy/index.html" target="_blank">HHS Vulnerability Disclosure</a></li> </ul> </div> </div> </div> </section> </footer> </div> <script type="application/json" data-drupal-selector="drupal-settings-json">{"path":{"baseUrl":"\/","pathPrefix":"","currentPath":"node\/27385","currentPathIsAdmin":false,"isFront":false,"currentLanguage":"en"},"pluralDelimiter":"\u0003","suppressDeprecationErrors":true,"ajaxPageState":{"libraries":"eJx1kFGOwzAIRC_kjb_3NBaJqeMWGwtw29y-jtTutqvsz2jmjQCJ-B20z7ZiQQ-qaOriG8K7oVSgLxiyWV7UDUS5XnyU3oCmZ3SJOREGg-TTkL95gjPcP2FxBBt3CzHrwleUzXPFhck1EEgCbdXXlV8y9dr6TFlXjE57QzkN73_cEQvrvj5Xw2qHvRYg0kUQ63G_u_EU_a9trNkyj-lNDYufQdF1vUUNu_UngYI3lssDdIKSjQ","theme":"d9_subtheme","theme_token":null},"ajaxTrustedUrl":[],"gtm":{"tagId":null,"settings":{"data_layer":"dataLayer","include_classes":false,"allowlist_classes":"","blocklist_classes":"","include_environment":false,"environment_id":"","environment_token":""},"tagIds":["GTM-55CGV66V"]},"gtag":{"tagId":"G-ZZQ8H5G8ZX","otherIds":[],"events":[],"additionalConfigInfo":[]},"data":{"extlink":{"extTarget":true,"extTargetNoOverride":false,"extNofollow":false,"extNoreferrer":true,"extFollowNoOverride":false,"extClass":"ext","extLabel":"(link is external)","extImgClass":false,"extSubdomains":true,"extExclude":"","extInclude":"","extCssExclude":"","extCssExplicit":"","extAlert":true,"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\u0027s privacy policy when you follow the link.","mailtoClass":"mailto","mailtoLabel":"(link sends email)","extUseFontAwesome":false,"extIconPlacement":"append","extFaLinkClasses":"fa fa-external-link","extFaMailtoClasses":"fa fa-envelope-o","whitelistedDomains":[]}},"superfish":{"superfish-navigation":{"id":"superfish-navigation","sf":{"animation":{"opacity":"show","height":"show"},"speed":"fast"},"plugins":{"smallscreen":{"mode":"window_width","accordionButton":0,"title":"Navigation"},"supposition":true,"supersubs":true}}},"user":{"uid":0,"permissionsHash":"24b2a698a72e7a44d4f2d94d9880a6ba82bfbc8b8200dee23422144335254cbc"}}</script> <script src="/core/assets/vendor/jquery/jquery.min.js?v=3.7.1"></script> <script src="/core/assets/vendor/once/once.min.js?v=1.0.1"></script> <script src="/core/misc/drupalSettingsLoader.js?v=10.2.3"></script> <script src="/core/misc/drupal.js?v=10.2.3"></script> <script src="/core/misc/drupal.init.js?v=10.2.3"></script> <script src="/core/assets/vendor/tabbable/index.umd.min.js?v=6.2.0"></script> <script src="https://cdn.jsdelivr.net/npm/@uswds/uswds@3.3.0/dist/js/uswds.min.js"></script> <script src="/core/misc/progress.js?v=10.2.3"></script> <script src="/core/assets/vendor/loadjs/loadjs.min.js?v=4.2.0"></script> <script src="/core/misc/debounce.js?v=10.2.3"></script> <script src="/core/misc/announce.js?v=10.2.3"></script> <script src="/core/misc/message.js?v=10.2.3"></script> <script src="/core/misc/ajax.js?v=10.2.3"></script> <script src="/themes/custom/d9_subtheme/js/script.js?v=10.2.3"></script> <script src="/modules/contrib/extlink/extlink.js?v=10.2.3"></script> <script src="/modules/contrib/google_tag/js/gtm.js?srmo5o"></script> <script src="/modules/contrib/google_tag/js/gtag.js?srmo5o"></script> <script src="/modules/contrib/google_tag/js/gtag.ajax.js?srmo5o"></script> <script src="/libraries/drupal-superfish/superfish.js?srmo5o"></script> <script src="/libraries/drupal-superfish/jquery.hoverIntent.minified.js?srmo5o"></script> <script src="/libraries/drupal-superfish/sfsmallscreen.js?srmo5o"></script> <script src="/libraries/drupal-superfish/supposition.js?srmo5o"></script> <script src="/libraries/drupal-superfish/supersubs.js?srmo5o"></script> <script src="/modules/contrib/superfish/js/superfish.js?v=2.0"></script> </body> </html>