{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T00:58:54Z","timestamp":1791939534672,"version":"4.3.4"},"reference-count":31,"publisher":"Becaris Publishing Limited","issue":"9","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"published-print":{"date-parts":[[2023,9]]},"abstract":"<jats:p>\n            <jats:bold>Aim:<\/jats:bold>\n            To evaluate treatment effects of eteplirsen among patients with Duchenne muscular dystrophy.\n            <jats:bold>Methods:<\/jats:bold>\n            Using real-world claims and electronic medical record data, this retrospective comparative analysis assessed eteplirsen-treated and control cohorts matched by age, disease progression state, and pre-index period healthcare resource utilization. Poisson regression was used to evaluate eteplirsen effects on healthcare resource utilization outcomes.\n            <jats:bold>Results:<\/jats:bold>\n            Eteplirsen was associated with statistically significant reductions in rates of hospital encounters (31%), emergency room visits (31%), need for pulmonary management (33%), cardiac management (21%), tracheostomy (86%), and assisted ventilation (39%) versus the control group. Other assessed outcomes favored eteplirsen numerically but did not all reach statistical significance.\n            <jats:bold>Conclusion:<\/jats:bold>\n            Eteplirsen-treated patients had reduced rates of multiple healthcare resource utilization measures versus matched controls.\n          <\/jats:p>","DOI":"10.57264\/cer-2023-0086","type":"journal-article","created":{"date-parts":[[2023,8,23]],"date-time":"2023-08-23T13:42:45Z","timestamp":1692798165000},"source":"Crossref","is-referenced-by-count":14,"title":["Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA"],"prefix":"10.57264","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1102-0651","authenticated-orcid":false,"given":"Joel","family":"Iff","sequence":"first","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, MA\u00a002142, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6246-2292","authenticated-orcid":false,"given":"Yi","family":"Zhong","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Menlo Park, CA\u00a094025, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edward","family":"Tuttle","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Menlo Park, CA\u00a094025, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deepshekhar","family":"Gupta","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Menlo Park, CA\u00a094025, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xander","family":"Paul","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Menlo Park, CA\u00a094025, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7511-6441","authenticated-orcid":false,"family":"Erik\u00a0Henricson","sequence":"additional","affiliation":[{"name":"UC Davis Medical Center, Sacramento, CA\u00a095817, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"36897","reference":[{"issue":"1","key":"e_1_3_7_3_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. 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