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It entailed a structured approach, involving multiple committees and boards. From its inception in 2014, the guidance underwent revisions incorporating insights from gene therapy studies, cardiac function research, and innovative clinical trial designs.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>The guidance provides a deeper understanding of DMD and its variants, focusing on patient engagement, diagnostic criteria, natural history, biomarkers, and clinical trials. It underscores patient-focused drug development, the significance of dystrophin as a biomarker, and the pivotal role of magnetic resonance imaging in assessing disease progression. Additionally, the guidance addresses cardiomyopathy\u2019s prominence in DMD and the burgeoning field of gene therapy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>The updated guidance offers a comprehensive understanding of DMD, emphasizing patient-centric approaches, innovative trial designs, and the importance of biomarkers. The focus on cardiomyopathy and gene therapy signifies the evolving realm of DMD research. It acts as a crucial roadmap for sponsors, potentially leading to improved treatments for DMD.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3233\/jnd-230219","type":"journal-article","created":{"date-parts":[[2024,2,16]],"date-time":"2024-02-16T10:45:25Z","timestamp":1708080325000},"page":"499-523","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":9,"title":["Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies \u2013 Developing Potential Treatments for the Entire Spectrum of Disease"],"prefix":"10.1177","volume":"11","author":[{"given":"Craig","family":"McDonald","sequence":"first","affiliation":[{"name":"University of California Davis Health","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric","family":"Camino","sequence":"additional","affiliation":[{"name":"Parent Project Muscular Dystrophy, Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rafael","family":"Escandon","sequence":"additional","affiliation":[{"name":"DGBI Consulting, LLC, Bainbridge Island, Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard S.","family":"Finkel","sequence":"additional","affiliation":[{"name":"DGBI Consulting, LLC, Bainbridge Island, Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ryan","family":"Fischer","sequence":"additional","affiliation":[{"name":"Parent Project Muscular Dystrophy, Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kevin","family":"Flanigan","sequence":"additional","affiliation":[{"name":"Center for Experimental Neurotherapeutics","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pat","family":"Furlong","sequence":"additional","affiliation":[{"name":"Parent Project Muscular Dystrophy, Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rose","family":"Juhasz","sequence":"additional","affiliation":[{"name":"Nationwide Children\u2019s Hospital, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ann S.","family":"Martin","sequence":"additional","affiliation":[{"name":"Parent Project Muscular Dystrophy, Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chet","family":"Villa","sequence":"additional","affiliation":[{"name":"Trinity Health Michigan, Grand Rapids, MI, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H. 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