{"id":305065,"date":"2025-08-05T00:00:00","date_gmt":"2025-08-05T00:00:00","guid":{"rendered":"http:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfcg0003-2024-biopharma-muscular-dystrophy-landscape-forecast-niche-rare-disease-landscape\/"},"modified":"2026-03-31T10:25:30","modified_gmt":"2026-03-31T10:25:30","slug":"nrlfcg0003-2025-biopharma-muscular-dystrophy-landscape-forecast-niche-rare-disease-landscape-forecast-us-eu5","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfcg0003-2025-biopharma-muscular-dystrophy-landscape-forecast-niche-rare-disease-landscape-forecast-us-eu5\/","title":{"rendered":"Muscular Dystrophy &#8211; Landscape &#038; Forecast &#8211; Niche &#038; Rare Disease Landscape &#038; Forecast (US\/EU5)"},"content":{"rendered":"<p>Muscular dystrophy (<abbr data-abbreviation-entity=\"7274\" title=\"muscular dystrophy\">MD<\/abbr>) is a spectrum of genetic disorders characterized by muscle weakness that, in severe forms, can lead to loss of ambulation and early mortality. The standard treatment for <abbr data-abbreviation-entity=\"7274\" title=\"muscular dystrophy\">MD<\/abbr> is glucocorticoids, which can delay the loss of ambulation. For Duchenne muscular dystrophy (<abbr data-abbreviation-entity=\"5961\" title=\"Duchenne muscular dystrophy\">DMD<\/abbr>), the most common childhood-onset form, the conditional approvals of disease-modifying therapies (Sarepta\u2019s exon-skipping therapies Exondys 51, Vyondys 53, and Amondys 45; NS Pharma\u2019s Viltepso) reflect uncertainty about these drugs\u2019 efficacy. However, Sarepta\u2019s gene therapy Elevidys and Catalyst \/ Santhera\u2019s novel corticosteroid Agamree are expanding the treatment options by offering better efficacy. Nevertheless, treatments are still needed that can meaningfully delay or halt the progressive muscle degeneration associated with the various forms of <abbr data-abbreviation-entity=\"7274\" title=\"muscular dystrophy\">MD<\/abbr>. Many therapies with diverse mechanisms of action are in development for <abbr data-abbreviation-entity=\"5961\" title=\"Duchenne muscular dystrophy\">DMD<\/abbr> and other forms of <abbr data-abbreviation-entity=\"7274\" title=\"muscular dystrophy\">MD<\/abbr>; some of these therapies will help fulfill treatment needs.<\/p>\n<p><strong>Question answered<\/strong><\/p>\n<ul class=\"round-bullets\">\n<li>What are the sizes of the key <abbr data-abbreviation-entity=\"7274\" title=\"muscular dystrophy\">MD<\/abbr> patient populations in the United States and <abbr data-abbreviation-entity=\"6092\" title=\"France, Germany, Italy, Spain, UK\">EU5<\/abbr>, and how will they change through 2034?<\/li>\n<li>How is the gene therapy Elevidys being integrated into the current treatment landscape for <abbr data-abbreviation-entity=\"5961\" title=\"Duchenne muscular dystrophy\">DMD<\/abbr>?<\/li>\n<li>What are the greatest unmet clinical needs in the treatment of <abbr data-abbreviation-entity=\"7274\" title=\"muscular dystrophy\">MD<\/abbr>, according to experts? How well will pipeline therapies address current treatment gaps?<\/li>\n<li>Which emerging therapies will launch by 2034, and what will be their commercial impact on the <abbr data-abbreviation-entity=\"7274\" title=\"muscular dystrophy\">MD<\/abbr> market through 2034?<\/li>\n<\/ul>\n<p><strong>Content highlights<\/strong><\/p>\n<ul class=\"round-bullets\">\n<li>Publication date: August 2025<\/li>\n<li>Geography: United States and <abbr title=\"France, Germany, Italy, Spain, UK\">EU5<\/abbr><\/li>\n<li>Primary research: Country-specific interviews with thought-leading neurologists; survey data collected for this and other Clarivate research<\/li>\n<li>Epidemiology: Diagnosed prevalent and drug-treated cases of <abbr title=\"Duchenne muscular dystrophy\">DMD<\/abbr>, diagnosed prevalent cases of <abbr title=\"Duchenne muscular dystrophy\">DMD<\/abbr> by exon-skipping pattern and ambulatory status, diagnosed prevalent cases of other major <abbr title=\"muscular dystrophy\">MD<\/abbr> subtypes such as Becker <abbr title=\"muscular dystrophy\">MD<\/abbr>, limb-girdle <abbr title=\"muscular dystrophy\">MD<\/abbr>, and myotonic dystrophy<\/li>\n<li>Forecast: Drug-level sales and patient share of key <abbr data-abbreviation-entity=\"7274\" title=\"muscular dystrophy\">MD<\/abbr> therapies in 2024 and 2034<\/li>\n<li>Drug treatments: Coverage of key current and late-phase emerging therapies<\/li>\n<\/ul>\n<p><strong>Product description<\/strong><\/p>\n<p>Niche &#038; Rare Disease Landscape &#038; Forecast offers comprehensive market intelligence with world-class epidemiology, keen insight into current and emerging therapies, and drug forecasts supported by detailed primary and secondary research, enabling you to:<\/p>\n<ul class=\"round-bullets\">\n<li>Optimize your long-term disease and development strategy.<\/li>\n<li>Quantify market potential for your pipeline assets and those of your competitors.<\/li>\n<li>Understand a disease from top to bottom, including key patient populations, the current and future therapeutic landscape, and the evolving market trajectory.<\/li>\n<li>Gauge the commercial outlook and impact of key market events.<\/li>\n<\/ul>\n","protected":false},"template":"","class_list":["post-305065","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-muscular-dystrophy","biopharma-therapy-areas-neurology","biopharma-geography-us","biopharma-date-2297"],"acf":[],"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/305065","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":10,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/305065\/revisions"}],"predecessor-version":[{"id":575756,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/305065\/revisions\/575756"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=305065"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}