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This post hoc analysis utilized data from a randomized, 2-period study of domagrozumab versus placebo in 120 ambulatory boys with DMD. DXA measures of lean body mass were obtained from the whole body (excluding head), arms, legs and appendicular skeleton at baseline and every 16\u00a0weeks. Treatment effects on DXA measures for domagrozumab versus placebo were assessed at Week 49. At Week 49, domagrozumab statistically significantly increased lean body mass versus placebo in the appendicular skeleton (<jats:italic>p<\/jats:italic>\u2009=\u20090.050) and arms (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001). The relationship between lean body mass at Week 49 and functional endpoints at Week 97 was evaluated. Changes in lean body mass at Week 49 in all regions except arms were significantly correlated with percent change from baseline in 4-stair climb (4SC) at Week 97. DXA-derived percent lean mass at Week 49 also correlated with 4SC and North Star Ambulatory Assessment at Week 97. These data indicate that whole-body DXA measures can be used as biomarkers for treatment effects and disease progression in patients with DMD, and warrant further investigation.<\/jats:p><jats:p><jats:italic>Trial registration<\/jats:italic>: ClinicalTrials.gov, NCT02310763; registered 8 December 2014.<\/jats:p>","DOI":"10.1038\/s41598-022-23072-5","type":"journal-article","created":{"date-parts":[[2022,11,5]],"date-time":"2022-11-05T11:03:31Z","timestamp":1667646211000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy"],"prefix":"10.1038","volume":"12","author":[{"given":"Sarah P.","family":"Sherlock","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Palmer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn R.","family":"Wagner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hoda Z.","family":"Abdel-Hamid","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cuixia","family":"Tian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean K.","family":"Mah","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michela","family":"Guglieri","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Russell J.","family":"Butterfield","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lawrence","family":"Charnas","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shannon","family":"Marraffino","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2022,11,5]]},"reference":[{"key":"23072_CR1","doi-asserted-by":"publisher","first-page":"13","DOI":"10.1038\/s41572-021-00248-3","volume":"7","author":"D Duan","year":"2021","unstructured":"Duan, D., Goemans, N., Takeda, S., Mercuri, E. & Aartsma-Rus, A. 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KRW is an employee of and holds stock in F. Hoffmann-La Roche Ltd. HZA-H has received research support from and served on advisory boards for Sarepta Therapeutics, Biogen, NS Pharma, and AveXis\/Novartis. CT has been site investigator for clinical trials with Pfizer, AveXis\/Novartis, Catabasis, WaVe, Roche, PTC Therapeutics, FibroGen, Capricor, Santhera, Sarepta Therapeutics, and Summit. JKM has received research support for clinical trials from Pfizer, Italfarmaco, Sarepta Therapeutics, Catabasis, Roche, Biogen, Novartis, NS Pharma, PTC Therapeutics, and ReveraGen; and consultant fees from PTC Therapeutics, Roche, and Biogen. FM is a member of the Pfizer Rare Disease Scientific Advisory Board; received grant support from Biogen and Sarepta Therapeutics; and received consultant fees and\/or speakers\u2019 honoraria from Novartis, Biogen, Roche, Dyne Therapeutics, and PTC Therapeutics. MG has served as chair for a ReveraGen study; collaborated on research with ReveraGen and Sarepta Therapeutics; acted as principal investigator for clinical trials sponsored by Pfizer, Italfarmaco, Santhera Therapeutics, ReveraGen, Dynacure, Roche, PTC Therapeutics, and Summit; participated in advisory boards for Pfizer, NS Pharma, and Dyne (consultancies through Newcastle University); and performed consultancy work (speaker) for Sarepta Therapeutics. RJB has received funding via contracts for clinical trials from AveXis\/Novartis, PTC Therapeutics, Sarepta Therapeutics, Pfizer, and Biogen; and has served on scientific advisory boards for Sarepta Therapeutics, Biogen, AveXis\/Novartis, and Pfizer.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"18762"}}