{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T18:35:36Z","timestamp":1790534136690,"version":"4.1.0"},"reference-count":50,"publisher":"SAGE Publications","issue":"1","license":[{"start":{"date-parts":[[2022,1,31]],"date-time":"2022-01-31T00:00:00Z","timestamp":1643587200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2022,1,31]],"date-time":"2022-01-31T00:00:00Z","timestamp":1643587200000},"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":["Nucleic Acid Therapeutics"],"published-print":{"date-parts":[[2022,2,1]]},"abstract":"<jats:p>\n                    The aim of this Phase 1\/2, 2-part, multicenter trial was to report clinical safety and efficacy of long-term golodirsen treatment among ambulatory patients with exon 53 skip-amenable Duchenne muscular dystrophy (DMD). Part 1 was a 12-week, randomized, double-blind, placebo-controlled, dose-titration study followed by 9-week safety review. Part 2 was a 168-week, open-label evaluation of golodirsen 30\u2009mg\/kg. Part 1 primary endpoint was safety. Part 2 primary endpoints were dystrophin protein expression and 6-minute walk test (6MWT); secondary endpoints were percent predicted forced vital capacity (FVC%p) and safety.\n                    <jats:italic toggle=\"yes\">Post hoc<\/jats:italic>\n                    ambulation analyses used mutation-matched external natural history controls. All patients from Part 1 (golodirsen,\n                    <jats:italic toggle=\"yes\">n<\/jats:italic>\n                    \u2009=\u20098; placebo,\n                    <jats:italic toggle=\"yes\">n<\/jats:italic>\n                    \u2009=\u20094) plus 13 additional patients entered Part 2; 23 completed the study. Adverse events were generally mild, nonserious, and unrelated to golodirsen, with no safety-related discontinuations or deaths. Golodirsen increased dystrophin protein (16.0-fold;\n                    <jats:italic toggle=\"yes\">P<\/jats:italic>\n                    \u2009&lt;\u20090.001) and exon skipping (28.9-fold;\n                    <jats:italic toggle=\"yes\">P<\/jats:italic>\n                    \u2009&lt;\u20090.001). At 3 years, 6MWT change from baseline was \u221299.0\u2009m for golodirsen-treated patients versus \u2212181.4\u2009m for external controls (\n                    <jats:italic toggle=\"yes\">P<\/jats:italic>\n                    \u2009=\u20090.067), and loss of ambulation occurred in 9% versus 26% (\n                    <jats:italic toggle=\"yes\">P<\/jats:italic>\n                    \u2009=\u20090.21). FVC%p declined 8.4% over 3 years in golodirsen-treated patients, comparing favorably with literature-reported rates. This study provides evidence for golodirsen biologic activity and long-term safety in a declining DMD population and suggests functional benefit versus external controls. Clinical Trial Registration number: NCT02310906.\n                  <\/jats:p>","DOI":"10.1089\/nat.2021.0043","type":"journal-article","created":{"date-parts":[[2021,11,17]],"date-time":"2021-11-17T17:11:51Z","timestamp":1637169111000},"page":"29-39","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":129,"title":["Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1\/2 Trial"],"prefix":"10.1177","volume":"32","author":[{"given":"Laurent","family":"Servais","sequence":"first","affiliation":[{"name":"I-Motion Institute, H\u00f4pital Armand Trousseau, Paris, France."},{"name":"Division of Child Neurology, Centre de R\u00e9f\u00e9rences des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Li\u00e8ge &amp; University of Li\u00e8ge, Li\u00e8ge, Belgium."},{"name":"MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, United Kingdom."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eugenio","family":"Mercuri","sequence":"additional","affiliation":[{"name":"Pediatric Neurology Unit, Universit\u00e0 Cattolica del Sacro Cuore Roma, Rome, Italy."},{"name":"Nemo Clinical Centre, Fondazione Policlinico Universitario A Gemelli IRCCS, Rome, Italy."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Volker","family":"Straub","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michela","family":"Guglieri","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andreea M.","family":"Seferian","sequence":"additional","affiliation":[{"name":"I-Motion Institute, H\u00f4pital Armand Trousseau, Paris, France."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariacristina","family":"Scoto","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health, London, United Kingdom."},{"name":"National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniela","family":"Leone","sequence":"additional","affiliation":[{"name":"Nemo Clinical Centre, Fondazione Policlinico Universitario A Gemelli IRCCS, Rome, Italy."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erica","family":"Koenig","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Navid","family":"Khan","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashish","family":"Dugar","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaodong","family":"Wang","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Baoguang","family":"Han","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dan","family":"Wang","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9102-5232","authenticated-orcid":false,"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health, London, United Kingdom."},{"name":"National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"on behalf of the SKIP-NMD Study Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiara","family":"Brusa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Antonaci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Brogna","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Merli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mauro","family":"Monforte","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giulia","family":"Norcia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marika","family":"Pane","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gloria","family":"Ferrantini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"George","family":"Dickson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Morgan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valentina","family":"Sardone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cody","family":"Akana","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jay S.","family":"Charleston","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cody A.","family":"Desjardins","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saleh H.","family":"El-Husayni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diane E.","family":"Frank","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frederick J.","family":"Schnell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2022,1,31]]},"reference":[{"key":"e_1_3_4_2_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002891"},{"key":"e_1_3_4_3_2","doi-asserted-by":"publisher","DOI":"10.2147\/NDT.S93873"},{"key":"e_1_3_4_4_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30024-3"},{"key":"e_1_3_4_5_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01430-8"},{"key":"e_1_3_4_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0960-8966(02)00140-2"},{"key":"e_1_3_4_7_2","first-page":"117","article-title":"Survival in Duchenne muscular dystrophy","volume":"31","author":"Rall S","year":"2012","unstructured":"Rall S, and Grimm T. (2012). Survival in Duchenne muscular dystrophy. Acta Myol, 31:117\u2013120.","journal-title":"Acta Myol"},{"key":"e_1_3_4_8_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejpn.2016.07.020"},{"key":"e_1_3_4_9_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0218683"},{"key":"e_1_3_4_10_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e318225ab2e"},{"key":"e_1_3_4_11_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0052512"},{"key":"e_1_3_4_12_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21808"},{"key":"e_1_3_4_13_2","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.51046"},{"key":"e_1_3_4_14_2","doi-asserted-by":"publisher","DOI":"10.1002\/humu.23561"},{"key":"e_1_3_4_15_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-020-09800-9"},{"key":"e_1_3_4_16_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-150100"},{"key":"e_1_3_4_17_2","doi-asserted-by":"publisher","DOI":"10.1002\/humu.20918"},{"key":"e_1_3_4_18_2","doi-asserted-by":"publisher","DOI":"10.2147\/DDDT.S97635"},{"key":"e_1_3_4_19_2","volume-title":"Vyondys 53 [package insert]","unstructured":"Vyondys 53 [package insert]. Sarepta Therapeutics, Inc., Cambridge, MA, 2020."},{"key":"e_1_3_4_20_2","volume-title":"Exondys 51 [package insert]","unstructured":"Exondys 51 [package insert]. Sarepta Therapeutics, Inc., Cambridge, MA, 2020."},{"key":"e_1_3_4_21_2","volume-title":"Viltepso [package insert]","unstructured":"Viltepso [package insert]. NS Pharma, Inc., Paramus, NJ, 2020."},{"key":"e_1_3_4_22_2","volume-title":"Amondys 45 [package insert]","unstructured":"Amondys 45 [package insert]. Sarepta Therapeutics, Inc., Cambridge, MA, 2021."},{"key":"e_1_3_4_23_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000009233"},{"key":"e_1_3_4_24_2","doi-asserted-by":"publisher","DOI":"10.1186\/s40478-020-01106-1"},{"key":"e_1_3_4_25_2","doi-asserted-by":"publisher","DOI":"10.1186\/s40478-020-01106-1"},{"key":"e_1_3_4_26_2","doi-asserted-by":"publisher","DOI":"10.1097\/MD.0000000000015858"},{"key":"e_1_3_4_27_2","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23982"},{"key":"e_1_3_4_28_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27347"},{"key":"e_1_3_4_29_2","doi-asserted-by":"publisher","DOI":"10.1096\/fj.12-224170"},{"key":"e_1_3_4_30_2","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2013.4908"},{"key":"e_1_3_4_31_2","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25951"},{"key":"e_1_3_4_32_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000005680"},{"key":"e_1_3_4_33_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-210643"},{"key":"e_1_3_4_34_2","unstructured":"Summit Therapeutics. Proof of concept study to assess activity and safety of C1100 SMT(ezutomid) in boys with Duchenne muscular dystrophy. https:\/\/clinicaltrials.gov\/ct2\/show\/results\/NCT02858362?view=results Accessed March9 2021."},{"key":"e_1_3_4_35_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2018.05.013"},{"key":"e_1_3_4_36_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-200548"},{"key":"e_1_3_4_37_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-180351"},{"key":"e_1_3_4_38_2","doi-asserted-by":"publisher","DOI":"10.1002\/ana.24555"},{"key":"e_1_3_4_39_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(15)60025-3"},{"key":"e_1_3_4_40_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.25089"},{"key":"e_1_3_4_41_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0003-9993(97)90001-0"},{"key":"e_1_3_4_42_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23808"},{"key":"e_1_3_4_43_2","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.22847"},{"key":"e_1_3_4_44_2","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.23172"},{"key":"e_1_3_4_45_2","doi-asserted-by":"publisher","DOI":"10.1097\/00002060-199509001-00003"},{"key":"e_1_3_4_46_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2018.07.004"},{"key":"e_1_3_4_47_2","doi-asserted-by":"publisher","DOI":"10.1097\/01241398-198803000-00002"},{"key":"e_1_3_4_48_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2019.02.002"},{"key":"e_1_3_4_49_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.chest.2020.04.043"},{"key":"e_1_3_4_50_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0204485"},{"key":"e_1_3_4_51_2","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2020.1264"}],"container-title":["Nucleic Acid Therapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1089\/nat.2021.0043","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1089\/nat.2021.0043","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1089\/nat.2021.0043","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T21:02:21Z","timestamp":1777669341000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1089\/nat.2021.0043"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,1,31]]},"references-count":50,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2022,2,1]]}},"alternative-id":["10.1089\/nat.2021.0043"],"URL":"https:\/\/doi.org\/10.1089\/nat.2021.0043","relation":{},"ISSN":["2159-3337","2159-3345"],"issn-type":[{"value":"2159-3337","type":"print"},{"value":"2159-3345","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,1,31]]}}}