{"id":405286,"date":"2019-09-24T00:00:00","date_gmt":"2019-09-24T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfcg0003-2019-biopharma-muscular-dystrophy-niche-rare-disease-landscape-forecast-us-eu5-2019\/"},"modified":"2026-03-31T10:46:19","modified_gmt":"2026-03-31T10:46:19","slug":"nrlfcg0003-2019-biopharma-muscular-dystrophy-niche-rare-disease-landscape-forecast-us-eu5-2019","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfcg0003-2019-biopharma-muscular-dystrophy-niche-rare-disease-landscape-forecast-us-eu5-2019\/","title":{"rendered":"Muscular Dystrophy | Niche &#038; Rare Disease Landscape &#038; Forecast | US\/EU5 | 2019"},"content":{"rendered":"<p>Muscular dystrophies (MDs) are a spectrum of genetic disorders characterized by muscle weakness that, in severe disease forms, can lead to loss of ambulation and early death. Duchenne muscular dystrophy (DMD) is the most common subtype with childhood-onset, while myotonic dystrophy is the most common subtype with adult-onset. The unmet need for effective treatments that can meaningfully delay or halt progressive muscle degeneration in DMD, as well as in other severe and moderately severe MDs, is high. Ataluren (PTC Therapeutics\u2019 Translarna), which targets DMD patients with nonsense dystrophin mutations, has been available in some European markets for DMD following its conditional approval by the EMA in 2014; and eteplirsen (Sarepta\u2019s Exondys 51), which targets DMD patients with dystrophin mutations amenable for skipping of exon-51, has been\u00a0available in the United States since 2016, when it was granted conditional approval for DMD. Though both drugs target the underlying cause of disease in DMD\u00a0and, thus, could modify its course, their efficacy as observed in clinical trials appears modest. We anticipate the launch of two new potentially disease-modifying therapies\u2014Sarepta Therapeutics\u2019 exon-skipping agents\u00a0golodirsen and casimersen\u2014and two nonspecific symptomatic therapies\u2014Santhera Pharmaceuticals\u2019 idebenone\u00a0and Italfarmaco SpA\u2019s givinostat\u2014during the 2017-2027 forecast period. Despite the launch of four new agents, the\u00a0unmet need for effective pharmacotherapies\u00a0for\u00a0DMD\u00a0will remain high.<\/p>\n","protected":false},"template":"","class_list":["post-405286","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-muscular-dystrophy","biopharma-geography-us","biopharma-date-931"],"acf":[],"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/405286","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report"}],"about":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/types\/report"}],"version-history":[{"count":2,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/405286\/revisions"}],"predecessor-version":[{"id":576603,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/405286\/revisions\/576603"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=405286"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}