{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T11:09:57Z","timestamp":1791284997692,"version":"4.1.0"},"reference-count":32,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2016,12,23]],"date-time":"2016-12-23T00:00:00Z","timestamp":1482451200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2017,4]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>\n                    <jats:italic>Introduction<\/jats:italic>\n                    : ACE\u2010031 is a fusion protein of activin receptor type IIB and IgG1\u2010Fc, which binds myostatin and related ligands. It aims to disrupt the inhibitory effect on muscle development and provide potential therapy for myopathies like Duchenne muscular dystrophy (DMD).\n                    <jats:italic>Methods<\/jats:italic>\n                    : ACE\u2010031 was administered subcutaneously every 2\u20134 weeks to DMD boys in a randomized, double\u2010blind, placebo\u2010controlled, ascending\u2010dose trial. The primary objective was safety evaluation. Secondary objectives included characterization of pharmacokinetics and pharmacodynamics.\n                    <jats:italic>Results<\/jats:italic>\n                    : ACE\u2010031 was not associated with serious or severe adverse events. The study was stopped after the second dosing regimen due to potential safety concerns of epistaxis and telangiectasias. A trend for maintenance of the 6\u2010minute walk test (6MWT) distance in the ACE\u2010031 groups compared with a decline in the placebo group (not statistically significant) was noted, as was a trend for increased lean body mass and bone mineral density (BMD) and reduced fat mass.\n                    <jats:italic>Conclusion<\/jats:italic>\n                    : ACE\u2010031 use demonstrated trends for pharmacodynamic effects on lean mass, fat mass, BMD, and 6MWT. Non\u2013muscle\u2010related adverse events contributed to the decision to discontinue the study. Myostatin inhibition is a promising therapeutic approach for DMD.\n                    <jats:italic>Muscle Nerve<\/jats:italic>\n                    <jats:bold>55<\/jats:bold>\n                    : 458\u2013464, 2017\n                  <\/jats:p>","DOI":"10.1002\/mus.25268","type":"journal-article","created":{"date-parts":[[2016,7,27]],"date-time":"2016-07-27T19:03:02Z","timestamp":1469646182000},"page":"458-464","source":"Crossref","is-referenced-by-count":227,"title":["Myostatin inhibitor ACE\u2010031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo\u2010controlled clinical trial"],"prefix":"10.1002","volume":"55","author":[{"given":"Craig","family":"Campbell","sequence":"first","affiliation":[{"name":"Pediatrics, Epidemiology and Clinical Neurological Sciences Western University London Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hugh J.","family":"McMillan","sequence":"additional","affiliation":[{"name":"Children's Hospital of Eastern Ontario University of Ottawa Ottawa Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean K.","family":"Mah","sequence":"additional","affiliation":[{"name":"Alberta Children's Hospital University of Calgary Calgary Alberta Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark","family":"Tarnopolsky","sequence":"additional","affiliation":[{"name":"McMaster University Medical Centre Hamilton Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn","family":"Selby","sequence":"additional","affiliation":[{"name":"British Columbia Children's Hospital University of British Columbia Vancouver British Columbia Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ty","family":"McClure","sequence":"additional","affiliation":[{"name":"Acceleron Pharma Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dawn M.","family":"Wilson","sequence":"additional","affiliation":[{"name":"Acceleron Pharma Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew L.","family":"Sherman","sequence":"additional","affiliation":[{"name":"Acceleron Pharma Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana","family":"Escolar","sequence":"additional","affiliation":[{"name":"Kennedy Krieger Institute Johns Hopkins Medical School Baltimore Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kenneth M.","family":"Attie","sequence":"additional","affiliation":[{"name":"Acceleron Pharma Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2016,12,23]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/0960-8966(91)90039-U"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1177\/0009922809347777"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0960-8966(02)00140-2"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.1038\/387083a0"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa040933"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1038\/ng0997-71"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pgen.0030079"},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.200207056"},{"key":"e_1_2_6_11_1","doi-asserted-by":"publisher","DOI":"10.1152\/ajpendo.00107.2003"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.151270098"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0505996102"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1242\/jcs.090365"},{"key":"e_1_2_6_15_1","doi-asserted-by":"publisher","DOI":"10.1038\/nature01154"},{"key":"e_1_2_6_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.10385"},{"key":"e_1_2_6_17_1","doi-asserted-by":"publisher","DOI":"10.1242\/jcs.02482"},{"key":"e_1_2_6_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21743"},{"key":"e_1_2_6_19_1","doi-asserted-by":"publisher","DOI":"10.1152\/japplphysiol.00866.2009"},{"key":"e_1_2_6_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21944"},{"key":"e_1_2_6_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajpath.2010.11.071"},{"key":"e_1_2_6_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23539"},{"key":"e_1_2_6_23_1","unstructured":"AttieKM YangY WilsonDM HaltomE ShermanML.Increased lean mass and muscle volume in healthy post\u2010menopausal women treated with ACE\u2010031 (soluble activin type IIB receptor) an inhibitor of myostatin and other negative regulators of muscle. 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