{"id":587,"date":"2010-08-19T14:43:37","date_gmt":"2010-08-19T18:43:37","guid":{"rendered":"http:\/\/sciencebusiness.technewslit.com\/?p=587"},"modified":"2010-08-19T14:43:37","modified_gmt":"2010-08-19T18:43:37","slug":"fda-grants-orphan-status-for-neuromuscular-drug","status":"publish","type":"post","link":"https:\/\/technewslit.com\/sciencebusiness\/?p=587","title":{"rendered":"FDA Grants Orphan Status for Neuromuscular Drug"},"content":{"rendered":"<p><a href=\"http:\/\/www.acceleronpharma.com\/\">Acceleron Pharma, Inc.<\/a>, a biopharmaceutical company in Cambridge, Massachusetts, today said the U.S. Food and Drug Administration (FDA) granted ACE-031, its treatment for Duchenne Muscular Dystrophy (DMD), with an orphan drug designation.<\/p>\n<p>DMD is a debilitating and fatal genetic disorder characterized by the progressive loss of muscle strength and function. The disease affects mainly boys and occurs in approximately 1 in every 3,500 live male births. Acceleron Pharma says ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting signaling through a cell surface receptor called activin receptor type IIB. ACE-031 is currently being studied in a phase 2 clinical trial of patients with DMD.<\/p>\n<p>The FDA designates drugs as orphan drugs to encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. Under the <a href=\"http:\/\/www.fda.gov\/forindustry\/developingproductsforrarediseasesconditions\/overview\/ucm119477.htm\">Orphan Drug Act<\/a>, the FDA may provide grant funding towards clinical trial costs, tax advantages, user-fee benefits, and seven years of market exclusivity in the United States following drug approval by the FDA.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Acceleron Pharma, Inc., a biopharmaceutical company in Cambridge, Massachusetts, today said the U.S. Food and Drug Administration (FDA) granted ACE-031, its treatment for Duchenne Muscular Dystrophy (DMD), with an orphan drug designation. DMD is a debilitating and fatal genetic disorder characterized by the progressive loss of muscle strength and function. The disease affects mainly boys [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[15,5],"tags":[22,27],"class_list":["post-587","post","type-post","status-publish","format-standard","hentry","category-products","category-regulations","tag-fda","tag-pharmaceuticals"],"_links":{"self":[{"href":"https:\/\/technewslit.com\/sciencebusiness\/index.php?rest_route=\/wp\/v2\/posts\/587","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/technewslit.com\/sciencebusiness\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/technewslit.com\/sciencebusiness\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/technewslit.com\/sciencebusiness\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/technewslit.com\/sciencebusiness\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=587"}],"version-history":[{"count":2,"href":"https:\/\/technewslit.com\/sciencebusiness\/index.php?rest_route=\/wp\/v2\/posts\/587\/revisions"}],"predecessor-version":[{"id":589,"href":"https:\/\/technewslit.com\/sciencebusiness\/index.php?rest_route=\/wp\/v2\/posts\/587\/revisions\/589"}],"wp:attachment":[{"href":"https:\/\/technewslit.com\/sciencebusiness\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=587"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/technewslit.com\/sciencebusiness\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=587"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/technewslit.com\/sciencebusiness\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=587"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}