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This review discusses several features that must be considered during trial design for neuromuscular diseases, as well as other rare diseases, to maximise the possibility of trial success using historic examples. These features include well-defined inclusion criteria, matching criteria, alternatives to placebo-controlled trials and the selection of trial endpoints. These features will be particularly important in the coming years as the investigation into innovative therapy approaches for neuromuscular diseases continues.<\/jats:p>","DOI":"10.3389\/fgene.2021.759994","type":"journal-article","created":{"date-parts":[[2021,12,7]],"date-time":"2021-12-07T14:12:13Z","timestamp":1638886333000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":5,"title":["Lessons Learned From Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation"],"prefix":"10.3389","volume":"12","author":[{"given":"Georgia","family":"Stimpson","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mary","family":"Chesshyre","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Giovanni","family":"Baranello","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1965","published-online":{"date-parts":[[2021,12,7]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"135","DOI":"10.1002\/mus.20586","article-title":"Entries in the Leiden Duchenne Muscular Dystrophy Mutation Database: An Overview of Mutation Types and Paradoxical Cases that Confirm the reading-frame Rule","volume":"34","author":"Aartsma-Rus","year":"2006","journal-title":"Muscle Nerve"},{"key":"B2","doi-asserted-by":"publisher","DOI":"10.1002\/cpt.2247","article-title":"Prognostic Factors and Treatment\u2010Effect Modifiers in Spinal Muscular Atrophy","author":"Baranello","year":"2021","journal-title":"Clin. 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