{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T09:01:10Z","timestamp":1790758870244,"version":"4.1.0"},"reference-count":41,"publisher":"SAGE Publications","issue":"4","license":[{"start":{"date-parts":[[2023,5,24]],"date-time":"2023-05-24T00:00:00Z","timestamp":1684886400000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2023,7,4]]},"abstract":"<jats:sec>\n                    <jats:title>BACKGROUND:<\/jats:title>\n                    <jats:p>Duchenne muscular dystrophy (DMD) is a neuromuscular disease stemming from dystrophin gene mutations. Lack of dystrophin leads to progressive muscle damage and replacement of muscle with fibrotic and adipose tissue. Pamrevlumab (FG-3019), a fully human monoclonal antibody that binds to connective tissue growth factor (CTGF), is in Phase III development for treatment of DMD and other diseases.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>METHODS:<\/jats:title>\n                    <jats:p>MISSION (Study 079; NCT02606136) was an open-label, Phase II, single-arm trial of pamrevlumab in 21 non-ambulatory patients with DMD (aged\u226512 years, receiving corticosteroids) who received 35-mg\/kg intravenous infusions every 2 weeks for 2 years. The primary endpoint was change from baseline in percent predicted forced vital capacity (ppFVC). Secondary endpoints included other pulmonary function tests, upper limb function and strength assessments, and changes in upper arm fat and fibrosis scores on magnetic resonance imaging.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>RESULTS:<\/jats:title>\n                    <jats:p>Fifteen patients completed the trial. Annual change from baseline (SE) in ppFVC was \u20134.2 (0.7) (95% CI \u20135.5, \u20132.8). Rate of decline in ppFVC in pamrevlumab-treated patients was slower than observed in historical published trials of non-ambulatory patients. MISSION participants experienced slower-than-anticipated muscle function declines compared with natural history and historical published trials of non-ambulatory patients with DMD. Pamrevlumab was well-tolerated. Treatment-emergent adverse events were mild to moderate, and none led to study discontinuation.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>CONCLUSIONS:<\/jats:title>\n                    <jats:p>nti-CTGF therapy with pamrevlumab represents a potential treatment for DMD. The lack of internal control group limits the results.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3233\/jnd-230019","type":"journal-article","created":{"date-parts":[[2023,5,26]],"date-time":"2023-05-26T13:25:55Z","timestamp":1685107555000},"page":"685-699","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":25,"title":["Pamrevlumab, a Fully Human Monoclonal Antibody Targeting Connective Tissue Growth Factor, for Non-Ambulatory Patients with Duchenne Muscular Dystrophy"],"prefix":"10.1177","volume":"10","author":[{"given":"Anne M.","family":"Connolly","sequence":"first","affiliation":[{"name":"Nationwide Children\u2019s Hospital, Ohio State University College of Medicine, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig M.","family":"Zaidman","sequence":"additional","affiliation":[{"name":"Department of Neurology, Washington University at St. Louis, St. Louis, MO, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John F.","family":"Brandsema","sequence":"additional","affiliation":[{"name":"Division of Neurology, The Children\u2019s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Han C.","family":"Phan","sequence":"additional","affiliation":[{"name":"Rare Disease Research, LLC, Atlanta, GA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cuixia","family":"Tian","sequence":"additional","affiliation":[{"name":"Division of Neurology, Cincinnati Children\u2019s Hospital Medical Center, Cincinnati, OH, USA"},{"name":"Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xueping","family":"Zhang","sequence":"additional","affiliation":[{"name":"FibroGen, Inc., San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jack","family":"Li","sequence":"additional","affiliation":[{"name":"FibroGen, Inc., San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark D.","family":"Eisner","sequence":"additional","affiliation":[{"name":"FibroGen, Inc., San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ewa","family":"Carrier","sequence":"additional","affiliation":[{"name":"FibroGen, Inc., San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2023,5,24]]},"reference":[{"key":"ref001","doi-asserted-by":"publisher","DOI":"10.1186\/s13073-016-0316-x"},{"key":"ref002","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-021-00248-3"},{"key":"ref003","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(87)90579-4"},{"issue":"6","key":"ref004","first-page":"835","volume":"45","author":"Den Dunnen JT","year":"1989","journal-title":"Am J Hum Genet"},{"key":"ref005","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002337"},{"key":"ref006","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.23172"},{"key":"ref007","doi-asserted-by":"publisher","DOI":"10.1002\/mus.25089"},{"key":"ref008","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)32160-8"},{"key":"ref009","doi-asserted-by":"publisher","DOI":"10.3233\/JND-210665"},{"key":"ref010","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000009233"},{"key":"ref011","doi-asserted-by":"publisher","DOI":"10.3233\/JND-210643"},{"key":"ref012","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23982"},{"key":"ref013","doi-asserted-by":"publisher","DOI":"10.1002\/ana.24555"},{"key":"ref014","doi-asserted-by":"publisher","DOI":"10.1097\/MD.0000000000015858"},{"key":"ref015","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2020.1264"},{"key":"ref016","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-021-01512-2"},{"key":"ref017","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27347"},{"key":"ref018","doi-asserted-by":"publisher","DOI":"10.1002\/path.2952"},{"key":"ref019","doi-asserted-by":"publisher","DOI":"10.1186\/1755-1536-5-S1-S24"},{"key":"ref020","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2007.09.043"},{"key":"ref021","doi-asserted-by":"publisher","DOI":"10.1016\/s0960-8966(02)00071-8"},{"key":"ref022","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2613.2010.00750.x"},{"key":"ref023","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddt352"},{"key":"ref024","doi-asserted-by":"publisher","DOI":"10.1002\/jor.24337"},{"key":"ref025","doi-asserted-by":"publisher","DOI":"10.1016\/j.matbio.2019.01.002"},{"key":"ref026","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddw090"},{"key":"ref027","doi-asserted-by":"publisher","DOI":"10.1161\/HYPERTENSIONAHA.114.03279"},{"key":"ref028","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacbts.2018.10.007"},{"key":"ref029","doi-asserted-by":"publisher","DOI":"10.1016\/j.yjmcc.2015.11.003"},{"key":"ref030","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.201703-0507WS"},{"key":"ref031","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.12213"},{"key":"ref032","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.14361"},{"key":"ref033","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2015.07.009"},{"key":"ref034","doi-asserted-by":"publisher","DOI":"10.1002\/nbm.2851"},{"key":"ref035","doi-asserted-by":"publisher","DOI":"10.2217\/bmm-2018-0125"},{"key":"ref036","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2019.02.002"},{"key":"ref037","unstructured":"FDA\nDuchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment Guidance for Industry, [cited Jun 22]. Available from. 2022 https:\/\/www.fda.gov\/regulatory-information\/search-fda-guidance-documents\/duchenne-muscular-dystrophy-and-related-dystrophinopathies-developing-drugs-treatment-guidance."},{"key":"ref038","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2016.12.014"},{"key":"ref039","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0199223"},{"key":"ref040","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0113999"},{"key":"ref041","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(22)00125-9"}],"container-title":["Journal of Neuromuscular Diseases"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.3233\/JND-230019","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.3233\/JND-230019","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.3233\/JND-230019","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T23:52:28Z","timestamp":1777506748000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.3233\/JND-230019"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,5,24]]},"references-count":41,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2023,7,4]]}},"alternative-id":["10.3233\/JND-230019"],"URL":"https:\/\/doi.org\/10.3233\/jnd-230019","relation":{},"ISSN":["2214-3599","2214-3602"],"issn-type":[{"value":"2214-3599","type":"print"},{"value":"2214-3602","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,5,24]]}}}