{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,24]],"date-time":"2026-06-24T12:55:01Z","timestamp":1782305701912,"version":"3.54.5"},"reference-count":67,"publisher":"Becaris Publishing Limited","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"abstract":"<jats:p>\n            <jats:bold>Aim:<\/jats:bold>\n            Phosphorodiamidate morpholino oligomers (PMOs) are exon-skipping therapies administered through once-weekly intravenous infusions used to treat Duchenne muscular dystrophy (DMD). This study assessed treatment patterns among patients with DMD receiving PMOs using administrative claims data while accounting for limitations in claims data for these therapies.\n            <jats:bold>Materials &amp; methods:<\/jats:bold>\n            This study used Inovalon\n            <jats:sup>\u00ae<\/jats:sup>\n            public and private closed claims data (1 June 2016\u201331 March 2024). Male patients with \u22651 claim for a PMO approved for DMD in the US (eteplirsen, casimersen, golodirsen and viltolarsen) were included. Index date was the first PMO claim. All available follow-up data were used to assess continuous PMO claims coverage, \u226560-day and \u226530-day gaps in PMO claims and PMO re-initiation after a gap. Adherence during 1\u00a0year after index was measured using proportion of days covered (PDC). Treatment patterns were also assessed in patients stratified by baseline algorithm-defined nonambulatory status (inferred from claims).\n            <jats:bold>Results:<\/jats:bold>\n            Among 397 patients included, median (IQR) follow-up time was 788 (484, 1109) days. Gaps in PMO claims coverage occurred in 190 (47.9%) and 254 (64.0%) patients using \u226560-day and \u226530-day gaps, respectively, among whom 110 (57.9%) and 176 (69.3%) had PMO re-initiation. Using \u226560-day and \u226530-day gap lengths, median (IQR) time to first gap in PMO claims was 25.5 (22.3, 32.9) months and 13.5 (10.2, 17.7) months, respectively and median (IQR) time to PMO re-initiation (not including gap time) was 4.4 (2.8, 8.7) months and 2.5 (1.7, 3.2) months. Median (IQR) PDC was 78.8% (38.8, 94.0) during 1\u00a0year after index. PMO treatment patterns were generally similar in patients stratified by algorithm-defined nonambulatory status.\n            <jats:bold>Conclusion:<\/jats:bold>\n            In an analysis of administrative claims data, adherence to PMO treatment for DMD was high. For patients with a gap in PMO claims, most subsequently re-initiated treatment, indicating lower discontinuation rates than previously reported.\n          <\/jats:p>","DOI":"10.57264\/cer-2025-0037","type":"journal-article","created":{"date-parts":[[2025,7,18]],"date-time":"2025-07-18T13:47:51Z","timestamp":1752846471000},"source":"Crossref","is-referenced-by-count":3,"title":["Real-world phosphorodiamidate morpholino oligomer treatment patterns in Duchenne muscular dystrophy: a claims-based analysis"],"prefix":"10.57264","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3474-6874","authenticated-orcid":false,"given":"Shannon","family":"Grabich","sequence":"first","affiliation":[{"name":"Sarepta Therapeutics, Inc, Cambridge, MA 02142 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian","family":"Ung","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc, Cambridge, MA 02142 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0158-9905","authenticated-orcid":false,"given":"Aalok","family":"Nadkar","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc, Cambridge, MA 02142 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5439-7344","authenticated-orcid":false,"given":"Kathryn","family":"DeYoung","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc, Cambridge, MA 02142 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4067-8962","authenticated-orcid":false,"given":"James","family":"Signorovitch","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc, Boston, MA 02199, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3065-3956","authenticated-orcid":false,"given":"Aravindhan","family":"Veerapandiyan","sequence":"additional","affiliation":[{"name":"University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR 72202, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"36897","published-online":{"date-parts":[[2025,7,18]]},"reference":[{"issue":"10","key":"e_1_3_10_3_2","doi-asserted-by":"crossref","first-page":"18168","DOI":"10.3390\/molecules201018168","article-title":"Duchenne muscular dystrophy: from diagnosis to therapy","volume":"20","author":"Falzarano MS","year":"2015","unstructured":"Falzarano MS, Scotton C, Passarelli C, Ferlini A. Duchenne muscular dystrophy: from diagnosis to therapy. Molecules 20(10), 18168\u201318184 (2015).","journal-title":"Molecules"},{"issue":"1","key":"e_1_3_10_4_2","doi-asserted-by":"crossref","first-page":"13","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 7(1), 13 (2021).","journal-title":"Nat. Rev. Dis. Primers"},{"issue":"1","key":"e_1_3_10_5_2","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1186\/s13023-020-01430-8","article-title":"Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis","volume":"15","author":"Crisafulli S","year":"2020","unstructured":"Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S, Trifir\u00f2 G. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis. Orphanet J. Rare Dis. 15(1), 141 (2020).","journal-title":"Orphanet J. Rare Dis."},{"issue":"21","key":"e_1_3_10_6_2","doi-asserted-by":"crossref","first-page":"1363","DOI":"10.1056\/NEJM198805263182104","article-title":"Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy","volume":"318","author":"Hoffman EP","year":"1988","unstructured":"Hoffman EP, Fischbeck KH, Brown RH Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy. N. Engl. J. Med. 318(21), 1363\u20131368 (1988).","journal-title":"N. Engl. J. Med."},{"issue":"3","key":"e_1_3_10_7_2","doi-asserted-by":"crossref","first-page":"1223","DOI":"10.1002\/j.2040-4603.2015.tb00638.x","article-title":"The dystrophin complex: structure, function, and implications for therapy","volume":"5","author":"Gao QQ","year":"2015","unstructured":"Gao QQ, McNally EM. The dystrophin complex: structure, function, and implications for therapy. Compr. Physiol. 5(3), 1223\u20131239 (2015).","journal-title":"Compr. Physiol."},{"issue":"6","key":"e_1_3_10_8_2","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1016\/0092-8674(87)90579-4","article-title":"Dystrophin: the protein product of the Duchenne muscular dystrophy locus","volume":"51","author":"Hoffman EP","year":"1987","unstructured":"Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 51(6), 919\u2013928 (1987).","journal-title":"Cell"},{"key":"e_1_3_10_9_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-230220"},{"issue":"7","key":"e_1_3_10_10_2","doi-asserted-by":"crossref","first-page":"1241","DOI":"10.3390\/genes13071241","article-title":"Current outline of exon skipping trials in Duchenne muscular dystrophy","volume":"13","author":"Eser G","year":"2022","unstructured":"Eser G, Topalo\u011flu H. Current outline of exon skipping trials in Duchenne muscular dystrophy. Genes (Basel) 13(7), 1241 (2022).","journal-title":"Genes (Basel)"},{"key":"e_1_3_10_11_2","doi-asserted-by":"publisher","DOI":"10.3390\/jcm10040820"},{"issue":"2","key":"e_1_3_10_12_2","doi-asserted-by":"crossref","first-page":"S343","DOI":"10.3233\/JND-210682","article-title":"Exon-skipping in Duchenne muscular dystrophy","volume":"8","author":"Takeda S","year":"2021","unstructured":"Takeda S, Clemens PR, Hoffman EP. Exon-skipping in Duchenne muscular dystrophy. J. Neuromuscul. Dis. 8(Suppl. 2), S343\u2013S358 (2021).","journal-title":"J. Neuromuscul. Dis."},{"issue":"5","key":"e_1_3_10_13_2","doi-asserted-by":"crossref","first-page":"637","DOI":"10.1002\/ana.23982","article-title":"Eteplirsen for the treatment of Duchenne muscular dystrophy","volume":"74","author":"Mendell JR","year":"2013","unstructured":"Mendell JR, Rodino-Klapac LR, Sahenk Z Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann. Neurol. 74(5), 637\u2013647 (2013).","journal-title":"Ann. Neurol."},{"issue":"6","key":"e_1_3_10_14_2","doi-asserted-by":"crossref","first-page":"989","DOI":"10.3233\/JND-210643","article-title":"Open-label evaluation of eteplirsen in patients with Duchenne muscular dystrophy amenable to exon 51 skipping: PROMOVI trial","volume":"8","author":"McDonald CM","year":"2021","unstructured":"McDonald CM, Shieh PB, Abdel-Hamid HZ Open-label evaluation of eteplirsen in patients with Duchenne muscular dystrophy amenable to exon 51 skipping: PROMOVI trial. J. Neuromuscul. Dis. 8(6), 989\u20131001 (2021).","journal-title":"J. Neuromuscul. Dis."},{"issue":"1","key":"e_1_3_10_15_2","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1089\/nat.2021.0043","article-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 I\/II trial","volume":"32","author":"Servais L","year":"2022","unstructured":"Servais L, Mercuri E, Straub V 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 I\/II trial. Nucleic Acid Ther. 32(1), 29\u201339 (2022).","journal-title":"Nucleic Acid Ther."},{"issue":"8","key":"e_1_3_10_16_2","doi-asserted-by":"crossref","first-page":"982","DOI":"10.1001\/jamaneurol.2020.1264","article-title":"Safety, tolerability, and efficacy of viltolarsen in boys with Duchenne muscular dystrophy amenable to exon 53 skipping: a phase II randomized clinical trial","volume":"77","author":"Clemens PR","year":"2020","unstructured":"Clemens PR, Rao VK, Connolly AM Safety, tolerability, and efficacy of viltolarsen in boys with Duchenne muscular dystrophy amenable to exon 53 skipping: a phase II randomized clinical trial. JAMA Neurol. 77(8), 982\u2013991 (2020).","journal-title":"JAMA Neurol."},{"key":"e_1_3_10_17_2","doi-asserted-by":"crossref","first-page":"S102","DOI":"10.1016\/j.nmd.2022.07.248","article-title":"P.132 Casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: interim results from the phase III ESSENCE trial","volume":"32","author":"Iannaccone S","year":"2022","unstructured":"Iannaccone S, Phan H, Straub V P.132 Casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: interim results from the phase III ESSENCE trial. Neuromuscul. Disord. 32, S102 (2022).","journal-title":"Neuromuscul. Disord."},{"key":"e_1_3_10_18_2","volume-title":"EXONDYS 51 (eteplirsen) injection [package insert]","year":"2024","unstructured":"EXONDYS 51 (eteplirsen) injection [package insert]. 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Sarepta Therapeutics, Inc, MA, USA (July 2024)."},{"issue":"5","key":"e_1_3_10_22_2","doi-asserted-by":"crossref","first-page":"387","DOI":"10.1038\/s41573-023-00670-0","article-title":"Overcoming barriers to patient adherence: the case for developing innovative drug delivery systems","volume":"22","author":"Baryakova TH","year":"2023","unstructured":"Baryakova TH, Pogostin BH, Langer R, McHugh KJ. Overcoming barriers to patient adherence: the case for developing innovative drug delivery systems. Nat. Rev. Drug Discov. 22(5), 387\u2013409 (2023).","journal-title":"Nat. Rev. Drug Discov."},{"key":"e_1_3_10_23_2","doi-asserted-by":"crossref","first-page":"18","DOI":"10.7812\/TPP\/18-033","article-title":"The unmet challenge of medication nonadherence","volume":"22","author":"Kleinsinger F","year":"2018","unstructured":"Kleinsinger F. The unmet challenge of medication nonadherence. Perm. J. 22, 18\u2013033 (2018).","journal-title":"Perm. 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Pharm."},{"issue":"1","key":"e_1_3_10_38_2","doi-asserted-by":"crossref","first-page":"523","DOI":"10.1007\/s12325-024-03044-z","article-title":"Assessment of phosphorodiamidate morpholino oligomer treatment patterns for patients with Duchenne muscular dystrophy: a MarketScan claims analysis","volume":"42","author":"Klimchak AC","year":"2025","unstructured":"Klimchak AC, Signorovitch J, Innis B, Laverty CG, Gooch K. Assessment of phosphorodiamidate morpholino oligomer treatment patterns for patients with Duchenne muscular dystrophy: a MarketScan claims analysis. Adv. Ther. 42(1), 523\u2013536 (2025).","journal-title":"Adv. Ther."},{"issue":"1","key":"e_1_3_10_39_2","doi-asserted-by":"crossref","first-page":"e2353094","DOI":"10.1001\/jamanetworkopen.2023.53094","article-title":"Characteristics of patients receiving novel muscular dystrophy drugs in trials vs routine care","volume":"7","author":"Hong D","year":"2024","unstructured":"Hong D, Avorn J, Wyss R, Kesselheim AS. Characteristics of patients receiving novel muscular dystrophy drugs in trials vs routine care. JAMA Netw. Open 7(1), e2353094 (2024).","journal-title":"JAMA Netw. Open"},{"issue":"6","key":"e_1_3_10_40_2","doi-asserted-by":"crossref","first-page":"e0304099","DOI":"10.1371\/journal.pone.0304099","article-title":"Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials","volume":"19","author":"McDonald CM","year":"2024","unstructured":"McDonald CM, Signorovitch J, Mercuri E Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials. PLoS ONE 19(6), e0304099 (2024).","journal-title":"PLoS ONE"},{"issue":"3","key":"e_1_3_10_41_2","doi-asserted-by":"crossref","first-page":"184","DOI":"10.1002\/cns3.20076","article-title":"Access to novel therapies for Duchenne muscular dystrophy\u2014Insights from expert treating physicians","volume":"2","author":"Veerapandiyan A","year":"2024","unstructured":"Veerapandiyan A, Connolly AM, Mathews KD Access to novel therapies for Duchenne muscular dystrophy\u2014Insights from expert treating physicians. Ann. Child Neurol. Soc. 2(3), 184\u2013188 (2024).","journal-title":"Ann. Child Neurol. Soc."},{"key":"e_1_3_10_42_2","unstructured":"United Healthcare. Individual exchange medical benefit drug policy: viltepso\u00ae (viltolarsen). Effective 1 June 2024. https:\/\/www.uhcprovider.com\/content\/dam\/provider\/docs\/public\/policies\/comm-medical-drug\/viltepso.pdf (Accessed: 18 October 2024)."},{"key":"e_1_3_10_43_2","unstructured":"United Healthcare. Individual exchange medical benefit drug policy: amondys 45\u00ae (casimersen). Effective 1 May 2024. https:\/\/www.uhcprovider.com\/content\/dam\/provider\/docs\/public\/policies\/comm-medical-drug\/amondys.pdf (Accessed: 18 October 2024)."},{"key":"e_1_3_10_44_2","unstructured":"United Healthcare. Individual exchange medical benefit drug policy: exondys 51\u00ae (eteplirsen). Effective 1 May 2024. https:\/\/www.uhcprovider.com\/content\/dam\/provider\/docs\/public\/policies\/comm-medical-drug\/exondys-51-eteplirsen.pdf (Accessed: 18 October 2024)."},{"key":"e_1_3_10_45_2","unstructured":"United Healthcare. Individual exchange medical benefit drug policy: vyondys 53\u00ae (golodirsen). Effective 1 May 2024. https:\/\/www.uhcprovider.com\/content\/dam\/provider\/docs\/public\/policies\/comm-medical-drug\/vyondys-53.pdf (Accessed: 18 October 2024)."},{"key":"e_1_3_10_46_2","unstructured":"Cigna. Drug and biologic coverage policy: viltolarsen. 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