{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T04:15:11Z","timestamp":1791951311657,"version":"4.3.4"},"reference-count":51,"publisher":"SAGE Publications","issue":"6","license":[{"start":{"date-parts":[[2021,11,2]],"date-time":"2021-11-02T00:00:00Z","timestamp":1635811200000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JND"],"published-print":{"date-parts":[[2021,11,2]]},"abstract":"<jats:p>Background: Duchenne muscular dystrophy (DMD) is a rare x-linked recessive genetic disorder affecting 1 in every 5000\u201310000 [1, 2]. This disease leads to a variable but progressive sequential pattern of muscle weakness that eventually causes loss of important functional milestones such as the ability to walk. With promising drugs in development to ameliorate the effects of muscle weakness, these treatments must be associated with a clinically meaningful functional change. Objective: The objective of this analysis is to determine both distribution, minimal detectable change (MDC), and anchor-based, minimal clinically important difference, (MCID) of 12 month change values in standardized time function tests (TFT) used to monitor disease progression in DMD. Method: This is a retrospective analysis of prospectively collected data from a multi-center prospective natural history study with the Cooperative International Neuromuscular Research Group (CINRG). This study calculated MDC and MCID values for 3 commonly used timed function tests typically used to monitor disease progression; supine to stand (STS), 10 meter walk\/run (10MWT), and 4 stair climb (4SC). MDC used standard error of measurement (SEM) while MCID measurements used the Vignos scale as an anchor to determine clinical change in functional status. Results: All 3 TFT were significantly important clinical endpoints to detect MDC and MCID changes. MDC and MCID 12 month changes were significant in 10MWT (\u20130.138, \u20130.212), Supine to Stand (\u20130.026, \u20130.023) and 4 stair climb (\u20130.034, \u20130.035) with an effect size greater or close to 0.2. Conclusion: The 3 TFT are clinically meaningful endpoints used to establish change in DMD. MCID values were higher than MDC values indicating that an anchor-based approach using Vignos as a clinically meaningful loss of lower extremity abilities is appropriate to assess change in boys with DMD.<\/jats:p>","DOI":"10.3233\/jnd-210646","type":"journal-article","created":{"date-parts":[[2021,6,18]],"date-time":"2021-06-18T14:47:55Z","timestamp":1624027675000},"page":"939-948","source":"Crossref","is-referenced-by-count":31,"title":["The Minimal Clinical Important Difference (MCID) in Annual Rate of Change of Timed Function Tests in Boys with DMD"],"prefix":"10.1177","volume":"8","author":[{"given":"Tina","family":"Duong","sequence":"first","affiliation":[{"name":"Department of Neurology, Stanford University School of Medicine, Stanford, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"CINRG-DNHS Investigators","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Canbek","sequence":"additional","affiliation":[{"name":"Physical Therapy Department, Nova Southeastern University, Fort Lauderdale, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marisa","family":"Birkmeier","sequence":"additional","affiliation":[{"name":"Department of Health, Human Function, and Rehabilitation Sciences, The George Washington University School of Medicine and Health Sciences, Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leslie","family":"Nelson","sequence":"additional","affiliation":[{"name":"Department of Physical Therapy, University of Texas Southwestern Medical Center, Dallas, TX, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Siener","sequence":"additional","affiliation":[{"name":"Department of Neurology, Washington University, St. Louis, MO, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alicia","family":"Fernandez-Fernandez","sequence":"additional","affiliation":[{"name":"Physical Therapy Department, Nova Southeastern University, Fort Lauderdale, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erik","family":"Henricson","sequence":"additional","affiliation":[{"name":"University of California Davis Health, Department of Physical Medicine and Rehabilitation, Sacramento CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig M.","family":"McDonald","sequence":"additional","affiliation":[{"name":"University of California Davis Health, Department of Physical Medicine and Rehabilitation, Sacramento CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather","family":"Gordish-Dressman","sequence":"additional","affiliation":[{"name":"Children\u2019s National Medical Center, Department of Biostatistics, Washington DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","reference":[{"issue":"3","key":"10.3233\/JND-210646_ref1","doi-asserted-by":"crossref","first-page":"513","DOI":"10.1542\/peds.2014-2044","article-title":"Prevalence of Duchenne and Becker muscular dystrophies in the United States","volume":"135","author":"Romitti","year":"2015","journal-title":"Pediatrics"},{"issue":"79","key":"10.3233\/JND-210646_ref2","first-page":"1","article-title":"M","volume":"12","author":"Ryder","year":"2017","journal-title":"L., Armstrong N, Westwood M, de Kock S, Butt T, Jain M, Kelijnen J. 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