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The clinical classification of subjects into severe (type 1), intermediate (type 2), and mild (type 3) subtypes has proved useful both in enhancing communication among clinicians internationally and in forging the collaborative development of outcome measures for clinical trials. Ideally, clinical trial design in spinal muscular atrophy must take into account the spinal muscular atrophy type, patient age, severity-of-affection status, nature of the therapeutic approach, timing of the proposed intervention relative to disease progression, and relative homogeneity of the cohort to be studied. Following is an overview of the challenges and opportunities, current and future therapeutic strategies, and progress to date in clinical trials in spinal muscular atrophy.<\/jats:p>","DOI":"10.1177\/0883073807305665","type":"journal-article","created":{"date-parts":[[2007,8,29]],"date-time":"2007-08-29T20:32:29Z","timestamp":1188419549000},"page":"957-966","source":"Crossref","is-referenced-by-count":56,"title":["Perspectives on Clinical Trials in Spinal Muscular Atrophy"],"prefix":"10.1177","volume":"22","author":[{"given":"Kathryn J.","family":"Swoboda","sequence":"first","affiliation":[{"name":"Departments of Neurology and Pediatrics, University of Utah School of Medicine, Salt Lake City, Utah,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John T.","family":"Kissel","sequence":"additional","affiliation":[{"name":"Department of Neurology, Ohio State University School of Medicine, Columbus, 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