{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,4]],"date-time":"2026-09-04T22:26:41Z","timestamp":1788560801185,"version":"build-2803163510"},"reference-count":29,"publisher":"SAGE Publications","issue":"3","license":[{"start":{"date-parts":[[2024,4,11]],"date-time":"2024-04-11T00:00:00Z","timestamp":1712793600000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2024,4,11]],"date-time":"2024-04-11T00:00:00Z","timestamp":1712793600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2024,4,30]]},"abstract":"<jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>BACKGROUND:<\/jats:title>\n                    <jats:p>\n                      Duchenne muscular dystrophy (DMD) is a rare, degenerative, recessive X-linked neuromuscular disease. Mutations in the gene encoding dystrophin lead to the absence of functional dystrophin protein. Individuals living with DMD exhibit progressive muscle weakness resulting in loss of ambulation and limb function, respiratory insufficiency, and cardiomyopathy, with multiorgan involvement. Adeno-associated virus vector-mediated gene therapy designed to enable production of functional dystrophin protein is a new therapeutic strategy. Delandistrogene moxeparvovec (Sarepta Therapeutics, Cambridge, MA) is indicated for treatment of ambulatory pediatric patients aged 4 through 5 years with DMD who have an indicated mutation in the\n                      <jats:italic toggle=\"yes\">DMD<\/jats:italic>\n                      gene.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>OBJECTIVE:<\/jats:title>\n                    <jats:p>Evidence-based considerations for management of potential adverse events following gene therapy treatment for DMD are lacking in clinical literature. Our goal was to provide interdisciplinary consensus considerations for selected treatment-related adverse events (TRAEs) (vomiting, acute liver injury, myocarditis, and immune-mediated myositis) that may arise following gene therapy dosing with delandistrogene moxeparvovec.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>METHODS:<\/jats:title>\n                    <jats:p>An interdisciplinary panel of 12 specialists utilized a modified Delphi process to develop consensus considerations for the evaluation and management of TRAEs reported in delandistrogene moxeparvovec clinical studies. Panelists completed 2 Questionnaires prior to gathering for an in-person discussion. Consensus was defined as a majority (\u226558% ; 7\/12) of panelists either agreeing or disagreeing.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>RESULTS:<\/jats:title>\n                    <jats:p>Panelists agreed that the choice of baseline assessments should be informed by individual clinical indications, the treating provider\u2019s judgment, and prescribing information. Corticosteroid dosing for treatment of TRAEs should be optimized by considering individual risk versus benefit for each indication. In all cases involving patients with a confirmed TRAE, consultations with appropriate specialists were suggested.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>CONCLUSIONS:<\/jats:title>\n                    <jats:p>The Delphi Panel established consensus considerations for the evaluation and management of potential TRAEs for patients receiving delandistrogene moxeparvovec, including vomiting, acute liver injury, myocarditis, and immune-mediated myositis.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3233\/jnd-230185","type":"journal-article","created":{"date-parts":[[2024,4,12]],"date-time":"2024-04-12T11:36:34Z","timestamp":1712921794000},"page":"687-699","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":23,"title":["Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy"],"prefix":"10.1177","volume":"11","author":[{"given":"Craig M.","family":"Zaidman","sequence":"first","affiliation":[{"name":"Washington University School of Medicine and St. Louis Children\u2019s Hospital","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalie L.","family":"Goedeker","sequence":"additional","affiliation":[{"name":"Washington University School of Medicine and St. Louis Children\u2019s Hospital","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amal A.","family":"Aqul","sequence":"additional","affiliation":[{"name":"University of Texas Southwestern Medical Center","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Russell J.","family":"Butterfield","sequence":"additional","affiliation":[{"name":"University of Utah School of Medicine","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne M.","family":"Connolly","sequence":"additional","affiliation":[{"name":"The Abigail Wexner Research Institute","place":["USA"]},{"name":"The Ohio State University","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ronald G.","family":"Crystal","sequence":"additional","affiliation":[{"name":"Weill Cornell Medical College","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kara E.","family":"Godwin","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kan N.","family":"Hor","sequence":"additional","affiliation":[{"name":"Nationwide Children\u2019s Hospital","place":["USA"]},{"name":"The Ohio State University","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katherine D.","family":"Mathews","sequence":"additional","affiliation":[{"name":"University of Iowa Carver College of Medicine","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Crystal M.","family":"Proud","sequence":"additional","affiliation":[{"name":"Children\u2019s Hospital of the King\u2019s Daughters","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth","family":"Kula Smyth","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aravindhan","family":"Veerapandiyan","sequence":"additional","affiliation":[{"name":"University of Arkansas for Medical Sciences","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul B.","family":"Watkins","sequence":"additional","affiliation":[{"name":"University of North Carolina Institute for Drug Safety Sciences","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jerry R.","family":"Mendell","sequence":"additional","affiliation":[{"name":"The Abigail Wexner Research Institute","place":["USA"]},{"name":"The Ohio State University","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2024,4,11]]},"reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM198807073190103"},{"issue":"309","key":"e_1_3_5_3_2","first-page":"64","article-title":"Renal, hepatic and immune function indices in patients with Duchenne muscular dystrophy","author":"Kononets O","year":"2020","unstructured":"KononetsO, KaraievT, TkachenkoO, LichmanL. Renal, hepatic and immune function indices in patients with Duchenne muscular dystrophy. Georgian Med News. 2020;(309);64\u201371.","journal-title":"Georgian Med News"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0275803"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(03)00585-4"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30024-3"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1706910"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2021.814174"},{"key":"e_1_3_5_10_2","unstructured":"U.S. Food and Drug Administration (FDA). Approved cellular and gene therapy products 2023 [updated 05\/19\/2023]. Available from: https:\/\/www.fda.gov\/vaccines-blood-biologics\/cellular-gene-therapy-products\/approved-cellular-and-gene-therapy-products."},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"e_1_3_5_12_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(23)00313-7"},{"key":"e_1_3_5_13_2","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2020.1484"},{"key":"e_1_3_5_14_2","unstructured":"Sarepta Therapeutics Inc. SRP-9001 (delandistrogenemoxeparvovec) for Treatment of Duchenne Muscular Dystrophy. Presented at the Cellular Tissue and Gene Therapies Advisory Committee May 12 2023. Available from: https:\/\/www.fda.gov\/media\/168095\/download"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27955"},{"key":"e_1_3_5_16_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41573-019-0012-9"},{"key":"e_1_3_5_17_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMc2212912"},{"issue":"10","key":"e_1_3_5_18_2","first-page":"1","article-title":"The Delphi technique: Making sense of consensus","volume":"12","author":"Hsu C-C","year":"2007","unstructured":"HsuC-C, SandfordBA. The Delphi technique: Making sense of consensus. 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