{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,19]],"date-time":"2026-05-19T06:48:55Z","timestamp":1779173335848,"version":"3.51.4"},"reference-count":36,"publisher":"Springer Science and Business Media LLC","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2023,12,28]]},"DOI":"10.7759\/cureus.51237","type":"journal-article","created":{"date-parts":[[2023,12,28]],"date-time":"2023-12-28T17:36:00Z","timestamp":1703784960000},"source":"Crossref","is-referenced-by-count":6,"title":["AMONDYS 45 (Casimersen), a Novel Antisense Phosphorodiamidate Morpholino Oligomer: Clinical Considerations for Treatment in Duchenne Muscular Dystrophy"],"prefix":"10.7759","author":[{"given":"Megan  E","family":"Vasterling","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rebecca  J","family":"Maitski","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Brice  A","family":"Davis","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Julie  E","family":"Barnes","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rucha  A","family":"Kelkar","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rachel J","family":"Klapper","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hirni","family":"Patel","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shahab","family":"Ahmadzadeh","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sahar","family":"Shekoohi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alan D","family":"Kaye","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Giustino","family":"Varrassi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","reference":[{"key":"ref1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2014-2044","article-title":"Prevalence of Duchenne and Becker muscular dystrophies in the United States","volume":"135","author":"Romitti PA","year":"2015","unstructured":"Romitti PA, Zhu Y, Puzhankara S, et al.. Prevalence of Duchenne and Becker muscular dystrophies in the United States. Pediatrics. 2015, 135:513-21. 10.1542\/peds.2014-2044","journal-title":"Pediatrics"},{"key":"ref2","article-title":"Duchenne Muscular Dystrophy","author":"Venugopal V","year":"2023","unstructured":"Venugopal V, Pavlakis S. Duchenne Muscular Dystrophy. StatPearls [Internet]. StatPearls Publishing, Treasure Island (FL); 2023. http:\/\/www.ncbi.nlm.nih.gov\/books\/NBK482346\/:"},{"key":"ref3","unstructured":"AMONDYS 45 (Casimersen). Prescribing information. (2021). https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2021\/213026lbl.pdf."},{"key":"ref4","doi-asserted-by":"publisher","DOI":"10.1007\/s00223-023-01116-w","article-title":"Anti-RANKL therapy prevents glucocorticoid-induced bone loss and promotes muscle function in a mouse model of Duchenne muscular dystrophy","volume":"113","author":"Jayash SN","year":"2023","unstructured":"Jayash SN, Hamoudi D, Stephen LA, et al.. Anti-RANKL therapy prevents glucocorticoid-induced bone loss and promotes muscle function in a mouse model of Duchenne muscular dystrophy. Calcif Tissue Int. 2023, 113:449-68. 10.1007\/s00223-023-01116-w","journal-title":"Calcif Tissue Int"},{"key":"ref5","unstructured":"Dystrophinopathies Treatment & Management. (2023). https.\/\/emedicine.medscape.com\/article\/1173204-treatment?form=fpf."},{"key":"ref6","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-021-00248-3","article-title":"Duchenne muscular dystrophy","volume":"7","author":"Duan D","year":"2021","unstructured":"Duan D, Goemans N, Takeda S, Mercuri E, Aartsma-Rus A. Duchenne muscular dystrophy. Nat Rev Dis Primers. 2021, 7:13. 10.1038\/s41572-021-00248-3","journal-title":"Nat Rev Dis Primers"},{"key":"ref7","article-title":"Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials","volume":"8","author":"Shimizu-Motohashi Y","year":"2016","unstructured":"Shimizu-Motohashi Y, Miyatake S, Komaki H, Takeda S, Aoki Y. Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials. Am J Transl Res. 2016, 8:2471-89.","journal-title":"Am J Transl Res"},{"key":"ref8","doi-asserted-by":"publisher","DOI":"10.3390\/jpm9010001","article-title":"Restoring dystrophin expression in Duchenne muscular dystrophy: current status of therapeutic approaches","volume":"9","author":"Shimizu-Motohashi Y","year":"2019","unstructured":"Shimizu-Motohashi Y, Komaki H, Motohashi N, Takeda S, Yokota T, Aoki Y. Restoring dystrophin expression in Duchenne muscular dystrophy: current status of therapeutic approaches. J Pers Med. 2019, 9:1. 10.3390\/jpm9010001","journal-title":"J Pers Med"},{"key":"ref9","doi-asserted-by":"publisher","DOI":"10.1096\/fj.13-240044","article-title":"Muscle dysfunction and structural defects of dystrophin-null sapje mutant zebrafish larvae are rescued by ataluren treatment","volume":"28","author":"Li M","year":"2014","unstructured":"Li M, Andersson-Lendahl M, Sejersen T, Arner A. Muscle dysfunction and structural defects of dystrophin-null sapje mutant zebrafish larvae are rescued by ataluren treatment. FASEB J. 2014, 28:1593-9. 10.1096\/fj.13-240044","journal-title":"FASEB J"},{"key":"ref10","doi-asserted-by":"publisher","DOI":"10.2217\/cer-2019-0171","article-title":"Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study","volume":"9","author":"Mercuri E","year":"2020","unstructured":"Mercuri E, Muntoni F, Osorio AN, et al.. Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study. J Comp Eff Res. 2020, 9:341-60. 10.2217\/cer-2019-0171","journal-title":"J Comp Eff Res"},{"key":"ref11","doi-asserted-by":"publisher","DOI":"10.1111\/ncn3.12691","article-title":"Clinical development of novel therapies for Duchenne muscular dystrophy\u2014current and future","volume":"11","author":"Tominari T","year":"2023","unstructured":"Tominari T, Aoki Y. Clinical development of novel therapies for Duchenne muscular dystrophy\u2014current and future. Neurology and Clinical Neuroscience. 2023, 11:111-8. 10.1111\/ncn3.12691","journal-title":"Neurology and Clinical Neuroscience"},{"key":"ref12","doi-asserted-by":"publisher","DOI":"10.1007\/s12015-017-9792-7","article-title":"Creation of dystrophin expressing chimeric cells of myoblast origin as a novel stem cell based therapy for Duchenne muscular dystrophy","volume":"14","author":"Siemionow M","year":"2018","unstructured":"Siemionow M, Cwykiel J, Heydemann A, Garcia-Martinez J, Siemionow K, Szilagyi E. Creation of dystrophin expressing chimeric cells of myoblast origin as a novel stem cell based therapy for Duchenne muscular dystrophy. Stem Cell Rev Rep. 2018, 14:189-99. 10.1007\/s12015-017-9792-7","journal-title":"Stem Cell Rev Rep"},{"key":"ref13","doi-asserted-by":"publisher","DOI":"10.3389\/fphys.2023.1094359","article-title":"A new immunodeficient Duchenne muscular dystrophy rat model to evaluate engraftment after human cell transplantation","volume":"14","author":"Sato M","year":"2023","unstructured":"Sato M, Goto M, Yamanouchi K, Sakurai H. A new immunodeficient Duchenne muscular dystrophy rat model to evaluate engraftment after human cell transplantation. Front Physiol. 2023, 14:1094359. 10.3389\/fphys.2023.1094359","journal-title":"Front Physiol"},{"key":"ref14","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.80.15.4856","article-title":"Defective myoblasts identified in Duchenne muscular dystrophy","volume":"80","author":"Blau HM","year":"1983","unstructured":"Blau HM, Webster C, Pavlath GK. Defective myoblasts identified in Duchenne muscular dystrophy. Proc Natl Acad Sci U S A. 1983, 80:4856-60. 10.1073\/pnas.80.15.4856","journal-title":"Proc Natl Acad Sci U S A"},{"key":"ref15","doi-asserted-by":"publisher","DOI":"10.1016\/j.tips.2022.04.009","article-title":"Casimersen for the treatment of Duchenne muscular dystrophy","volume":"43","author":"Zakeri SE","year":"2022","unstructured":"Zakeri SE, Pradeep SP, Kasina V, Laddha AP, Manautou JE, Bahal R. Casimersen for the treatment of Duchenne muscular dystrophy. Trends Pharmacol Sci. 2022, 43:607-8. 10.1016\/j.tips.2022.04.009","journal-title":"Trends Pharmacol Sci"},{"key":"ref16","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2014.8","article-title":"Targeted exon skipping to correct exon duplications in the dystrophin gene","volume":"3","author":"Greer KL","year":"2014","unstructured":"Greer KL, Lochm\u00fcller H, Flanigan K, Fletcher S, Wilton SD. Targeted exon skipping to correct exon duplications in the dystrophin gene. Mol Ther Nucleic Acids. 2014, 3:e155. 10.1038\/mtna.2014.8","journal-title":"Mol Ther Nucleic Acids"},{"key":"ref17","article-title":"P.132 Casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: interim results from the Phase 3 ESSENCE trial","volume":"32, Supplement 1","author":"Iannaccone S","year":"2022","unstructured":"Iannaccone S, Phan H, Straub V, et al.. P.132 Casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: interim results from the Phase 3 ESSENCE trial. Neuromuscul Disord. 2022, 32, Supplement 1:102.","journal-title":"Neuromuscul Disord"},{"key":"ref18","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27347","article-title":"Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: a randomized, double-blind, placebo-controlled, dose-titration trial","volume":"64","author":"Wagner KR","year":"2021","unstructured":"Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB. Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: a randomized, double-blind, placebo-controlled, dose-titration trial. Muscle Nerve. 2021, 64:285-92. 10.1002\/mus.27347","journal-title":"Muscle Nerve"},{"key":"ref19","doi-asserted-by":"publisher","DOI":"10.1124\/dmd.121.000418","article-title":"Adverse drug reactions and toxicity of the Food and Drug Administration-approved antisense oligonucleotide drugs","volume":"50","author":"Alhamadani F","year":"2022","unstructured":"Alhamadani F, Zhang K, Parikh R, et al.. Adverse drug reactions and toxicity of the Food and Drug Administration-approved antisense oligonucleotide drugs. Drug Metab Dispos. 2022, 50:879-87. 10.1124\/dmd.121.000418","journal-title":"Drug Metab Dispos"},{"key":"ref20","unstructured":"An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy. (2023). https.\/\/clinicaltrials.gov\/ct2\/show\/NCT03532542."},{"key":"ref21","unstructured":"Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) (ESSENCE). (2023). https.\/\/clinicaltrials.gov\/ct2\/show\/NCT02500381."},{"key":"ref22","unstructured":"A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications. (2023). https.\/\/clinicaltrials.gov\/ct2\/show\/NCT04179409."},{"key":"ref23","doi-asserted-by":"publisher","DOI":"10.1021\/acs.jmedchem.3c01236","article-title":"Discovery of YSR734: A covalent HDAC inhibitor with cellular activity in acute myeloid leukemia and Duchenne muscular dystrophy","author":"Raouf YS","year":"2023","unstructured":"Raouf YS, Sedighi A, Geletu M, et al.. Discovery of YSR734: A covalent HDAC inhibitor with cellular activity in acute myeloid leukemia and Duchenne muscular dystrophy. J Med Chem. 2023, 10.1021\/acs.jmedchem.3c01236","journal-title":"J Med Chem"},{"key":"ref24","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-023-48982-w","article-title":"Mutational spectrum and phenotypic variability of Duchenne muscular dystrophy and related disorders in a Bangladeshi population","volume":"13","author":"Sarker S","year":"2023","unstructured":"Sarker S, Eshaque TB, Soorajkumar A, et al.. Mutational spectrum and phenotypic variability of Duchenne muscular dystrophy and related disorders in a Bangladeshi population. Sci Rep. 2023, 13:21547. 10.1038\/s41598-023-48982-w","journal-title":"Sci Rep"},{"key":"ref25","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2023.1261318","article-title":"A novel splicing mutation identified in a DMD patient: a case report","volume":"11","author":"Wen Y","year":"2023","unstructured":"Wen Y, Yang L, Shen G, Dai S, Wang J, Wang X. A novel splicing mutation identified in a DMD patient: a case report. Front Pediatr. 2023, 11:1261318. 10.3389\/fped.2023.1261318","journal-title":"Front Pediatr"},{"key":"ref26","doi-asserted-by":"publisher","DOI":"10.1242\/dmm.050395","article-title":"Longitudinal assessment of skeletal muscle functional mechanics in the DE50-MD dog model of Duchenne muscular dystrophy","volume":"16","author":"Riddell DO","year":"2023","unstructured":"Riddell DO, Hildyard JC, Harron RC, Taylor-Brown F, Kornegay JN, Wells DJ, Piercy RJ. Longitudinal assessment of skeletal muscle functional mechanics in the DE50-MD dog model of Duchenne muscular dystrophy. Dis Model Mech. 2023, 16:10.1242\/dmm.050395","journal-title":"Dis Model Mech"},{"key":"ref27","doi-asserted-by":"publisher","DOI":"10.3389\/fcell.2023.1329048","article-title":"Editorial: personalized medicine for neuromuscular disorders","volume":"11","author":"Bartoli M","year":"2023","unstructured":"Bartoli M, Bailey RM, Meyer K, Barth\u00e9l\u00e9my F. Editorial: personalized medicine for neuromuscular disorders. Front Cell Dev Biol. 2023, 11:1329048. 10.3389\/fcell.2023.1329048","journal-title":"Front Cell Dev Biol"},{"key":"ref28","doi-asserted-by":"publisher","DOI":"10.1016\/S0929-693X(23)00221-X","article-title":"Basic notions about gene therapy from the nucleic acid perspective and applications in a pediatric disease: Duchenne muscular dystrophy","volume":"30","author":"Richard I","year":"2023","unstructured":"Richard I. Basic notions about gene therapy from the nucleic acid perspective and applications in a pediatric disease: Duchenne muscular dystrophy. Arch Pediatr. 2023, 30:8S2-S11. 10.1016\/S0929-693X(23)00221-X","journal-title":"Arch Pediatr"},{"key":"ref29","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2023.1276144","article-title":"Detecting early signs in Duchenne muscular dystrophy: comprehensive review and diagnostic implications","volume":"11","author":"Mercuri E","year":"2023","unstructured":"Mercuri E, Pane M, Cicala G, Brogna C, Ciafaloni E. Detecting early signs in Duchenne muscular dystrophy: comprehensive review and diagnostic implications. Front Pediatr. 2023, 11:1276144. 10.3389\/fped.2023.1276144","journal-title":"Front Pediatr"},{"key":"ref30","doi-asserted-by":"publisher","DOI":"10.1007\/s43630-023-00506-1","article-title":"Antioxidant effects of LEDT in dystrophic muscle cells: involvement of PGC-1\u03b1 and UCP-3 pathways","author":"da Rocha GL","year":"2023","unstructured":"da Rocha GL, Guimar\u00e3es DS, da Cruz MV, et al.. Antioxidant effects of LEDT in dystrophic muscle cells: involvement of PGC-1\u03b1 and UCP-3 pathways. Photochem Photobiol Sci. 2023, 10.1007\/s43630-023-00506-1","journal-title":"Photochem Photobiol Sci"},{"key":"ref31","doi-asserted-by":"publisher","DOI":"10.3233\/JND-230094","article-title":"Factors associated with respiratory health and function in Duchenne muscular dystrophy: a systematic review and evidence grading","author":"Landfeldt E","year":"2023","unstructured":"Landfeldt E, Aleman A, Abner S, et al.. Factors associated with respiratory health and function in Duchenne muscular dystrophy: a systematic review and evidence grading. J Neuromuscul Dis. 2023, 1-33. 10.3233\/JND-230094","journal-title":"J Neuromuscul Dis"},{"key":"ref32","doi-asserted-by":"publisher","DOI":"10.3233\/JND-230161","article-title":"Considering the promise of Vamorolone for treating Duchenne muscular dystrophy","volume":"10","author":"Grounds MD","year":"2023","unstructured":"Grounds MD, Lloyd EM. Considering the promise of Vamorolone for treating Duchenne muscular dystrophy. J Neuromuscul Dis. 2023, 10:1013-30. 10.3233\/JND-230161","journal-title":"J Neuromuscul Dis"},{"key":"ref33","doi-asserted-by":"crossref","DOI":"10.1007\/s00198-023-06951-z","article-title":"Growth hormone and testosterone delay vertebral fractures in boys with muscular dystrophy on chronic glucocorticoids","author":"Loscalzo E","year":"2023","unstructured":"Loscalzo E, See J, Bharill S, et al.. Growth hormone and testosterone delay vertebral fractures in boys with muscular dystrophy on chronic glucocorticoids. Osteoporos Int. Published online October. 2023,","journal-title":"Osteoporos Int. Published online October"},{"key":"ref34","doi-asserted-by":"publisher","DOI":"10.1530\/EC-23-0245","article-title":"Is ongoing testosterone required after pubertal induction in Duchenne muscular dystrophy?","volume":"12","author":"Wood CL","year":"2023","unstructured":"Wood CL, Hollingsworth KG, Bokaie E, et al.. Is ongoing testosterone required after pubertal induction in Duchenne muscular dystrophy?. Endocr Connect. 2023, 12:10.1530\/EC-23-0245","journal-title":"Endocr Connect"},{"key":"ref35","unstructured":"Delandistrogene Moxeparvovec (Elevidys) for Duchenne Muscular Dystrophy. (2023). https.\/\/secure.medicalletter.org\/TML-article-1686d."},{"key":"ref36","article-title":"Growth hormone therapy for children with Duchenne muscular dystrophy and glucocorticoid induced short stature","volume":"5","author":"Lavi E","year":"2023","unstructured":"Lavi E, Cohen A, Dor T, Tsabari R, Zangen D. Growth hormone therapy for children with Duchenne muscular dystrophy and glucocorticoid induced short stature. J Endocr Soc. 2023, 5:A715.","journal-title":"J Endocr Soc"}],"container-title":["Cureus"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.cureus.com\/articles\/215610-amondys-45-casimersen-a-novel-antisense-phosphorodiamidate-morpholino-oligomer-clinical-considerations-for-treatment-in-duchenne-muscular-dystrophy","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,2,9]],"date-time":"2024-02-09T20:11:17Z","timestamp":1707509477000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.cureus.com\/articles\/215610-amondys-45-casimersen-a-novel-antisense-phosphorodiamidate-morpholino-oligomer-clinical-considerations-for-treatment-in-duchenne-muscular-dystrophy"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,12,28]]},"references-count":36,"URL":"https:\/\/doi.org\/10.7759\/cureus.51237","relation":{},"ISSN":["2168-8184"],"issn-type":[{"value":"2168-8184","type":"print"}],"subject":[],"published":{"date-parts":[[2023,12,28]]}}}