{"id":389790,"date":"2022-08-19T00:00:00","date_gmt":"2022-08-19T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfcg0003-2022-biopharma-muscular-dystrophy-niche-rare-disease-landscape-forecast-us-eu5-2022\/"},"modified":"2026-03-31T10:38:44","modified_gmt":"2026-03-31T10:38:44","slug":"nrlfcg0003-2022-biopharma-muscular-dystrophy-niche-rare-disease-landscape-forecast-us-eu5-2022","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfcg0003-2022-biopharma-muscular-dystrophy-niche-rare-disease-landscape-forecast-us-eu5-2022\/","title":{"rendered":"Muscular Dystrophy | Niche &#038; Rare Disease Landscape &#038; Forecast | US\/EU5 | 2022"},"content":{"rendered":"<p>Muscular dystrophy (MD) is a spectrum of genetic disorders characterized by muscle weakness that, in severe forms, can lead to loss of ambulation and early mortality. Treatments are needed that can meaningfully delay or halt the progressive muscle degeneration associated with Duchenne muscular dystrophy (DMD), the most common childhood-onset form, as well as other forms of\u00a0MD. The standard treatment for\u00a0MD\u00a0is glucocorticoids, which have proven effective in delaying the loss of ambulation. Recent conditional approvals of genotype-specific, disease-modifying therapies such as Sarepta\u2019s Exondys 51, Vyondys 53, and Amondys 45; Nippon Shinyaku\u2019s Viltolarsen; and PTC Therapeutics\u2019 Translarna have expanded treatment options for\u00a0DMD\u00a0patients, but there are lingering concerns about these drugs\u2019 clinical efficacy. Additionally, many therapies with diverse mechanisms of action are being developed to treat other forms of\u00a0MD, including limb-girdle\u00a0MD\u00a0and Becker\u00a0MD. Overall, there is still high unmet need for more-effective treatments for\u00a0DMD\u00a0and other forms of\u00a0MD.<\/p>\n<p><strong>QUESTIONS ANSWERED<\/strong><\/p>\n<ul>\n<li>What are the sizes of key\u00a0MD\u00a0patient populations in the United States and\u00a0EU5\u00a0countries, and how will the population change through 2031?<\/li>\n<li>What is the current treatment landscape in\u00a0MD, and where are the greatest unmet clinical needs according to experts?<\/li>\n<li>How well will pipeline therapies address current treatment gaps?<\/li>\n<li>Which emerging therapies will launch before 2031, and what will be their commercial impact on the\u00a0MD\u00a0market through 2031?<\/li>\n<\/ul>\n<p><strong>GEOGRAPHIES<\/strong><\/p>\n<p>United States,\u00a0EU5<\/p>\n<p><strong>PRIMARY<\/strong><strong>\u00a0<\/strong><strong>RESEARCH<\/strong><\/p>\n<p>Six country-specific interviews with thought-leading neurologists<\/p>\n<p>Supported by survey data collected for this study<\/p>\n<p><strong>EPIDEMIOLOGY<\/strong><\/p>\n<p>Diagnosed prevalent and drug-treated cases of DMD, diagnosed prevalent cases of DMD by exon-skipping pattern and ambulatory status, diagnosed prevalent cases of Becker MD, limb-girdle MD, facioscapulohumeral MD, and myotonic dystrophy.<\/p>\n<p><strong>FORECAST<\/strong><\/p>\n<p>Drug-level sales and patient shares of key muscular dystrophy therapies through 2031<\/p>\n<p><strong>EMERGING<\/strong><strong>\u00a0<\/strong><strong>THERAPIES<\/strong><\/p>\n<p>Phase III\/PR: 6 drugs; Phase I\/II: > 10 drugs; coverage of select early-phase and preclinical products.<\/p>\n<p><strong>Product\u00a0<\/strong><strong>Description<\/strong><\/p>\n<p>Niche &#038; Rare Disease Landscape &#038; Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.<\/p>\n","protected":false},"template":"","class_list":["post-389790","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-muscular-dystrophy","biopharma-geography-us","biopharma-date-960"],"acf":[],"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/389790","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\/389790\/revisions"}],"predecessor-version":[{"id":576218,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/389790\/revisions\/576218"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=389790"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}