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The appropriateness and validity of biomarkers and clinically meaningful endpoints and an understanding of disease progression rates all played essential roles in the levels of evidence for these drugs. Such tools are best developed through integration of clinical data. The siloing of clinical data for rare neuromuscular diseases represents a considerable barrier to achieving better care and novel therapies for patients living with neuromuscular diseases. We discuss a data\u2010sharing model implemented for DMD and urge cultural changes in the ways natural history and clinical trial data are collected and shared across all neuromuscular diseases in order to benefit the primary stakeholder, the patient. <jats:italic>Muscle Nerve<\/jats:italic> <jats:bold>57<\/jats:bold>: 16\u201319, 2018<\/jats:p>","DOI":"10.1002\/mus.25961","type":"journal-article","created":{"date-parts":[[2017,9,7]],"date-time":"2017-09-07T18:28:29Z","timestamp":1504808909000},"page":"16-19","source":"Crossref","is-referenced-by-count":0,"title":["Seeking a better landscape for therapy development in neuromuscular disorders"],"prefix":"10.1002","volume":"57","author":[{"given":"Jane","family":"Larkindale","sequence":"first","affiliation":[{"name":"Duchenne Regulatory Science Consortium Critical Path Institute 1730 East River Road, Tucson Arizona 85718 USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6724-6409","authenticated-orcid":false,"given":"John D.","family":"Porter","sequence":"additional","affiliation":[{"name":"Myotonic Dystrophy Foundation San Francisco California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2017,9,23]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMe1516564"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMp1605654"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s13311-015-0336-z"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jalz.2014.11.005"},{"key":"e_1_2_9_6_1","unstructured":"US Food and Drug Administration.2016.Guidance for industry: Qualification of biomarker\u2014total kidney volume in studies for treatment of autosomal dominant polycystic kidney disease. 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