{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,3]],"date-time":"2026-05-03T10:40:35Z","timestamp":1777804835688,"version":"3.51.4"},"reference-count":31,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2024,10,21]],"date-time":"2024-10-21T00:00:00Z","timestamp":1729468800000},"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":["J Child Neurol"],"published-print":{"date-parts":[[2025,2]]},"abstract":"<jats:p>Duchenne muscular dystrophy is a genetically based neuromuscular disorder characterized by progressive physical impairment and cardiomyopathy in children, leading to fatal cardiac or respiratory failure. Duchenne muscular dystrophy shares some overlapping clinical features with other disorders, complicating clinical differentiation. We hypothesized that some Duchenne muscular dystrophy patients may have a secondary neuromuscular disorders that could negatively skew data during pharmaceutical clinical trials and lead to incomplete treatment plans. Consecutive genetic panels on 353 patients were reviewed. Thirty-two (32; 9.1%) patients with Duchenne muscular dystrophy were identified. Three (3; 9.4%) were found to have at least 1 genetically confirmed secondary neuromuscular disorder. Overlooking these coexisting disorders could lead to unexpected treatment failures, potentially affecting medication efficacy in trials or commercial use. Secondary neuromuscular disorders should be considered in Duchenne muscular dystrophy patients before clinical trial enrollment or treatment planning, with expanded genetic testing, such as whole exome sequencing or whole genome sequencing, likely to reveal even more secondary disorders.<\/jats:p>","DOI":"10.1177\/08830738241284683","type":"journal-article","created":{"date-parts":[[2024,10,21]],"date-time":"2024-10-21T05:02:56Z","timestamp":1729486976000},"page":"83-90","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Genetic Panel Reveals Coexisting Neuromuscular Disorders in Patients With Duchenne Muscular Dystrophy"],"prefix":"10.1177","volume":"40","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-0189-4108","authenticated-orcid":false,"given":"Carter","family":"Butson","sequence":"first","affiliation":[{"name":"Texas College of Osteopathic Medicine, UNT Health Science Center, Fort Worth, TX, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nedeke","family":"Ntekim","sequence":"additional","affiliation":[{"name":"Texas College of Osteopathic Medicine, UNT Health Science Center, Fort Worth, TX, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stephanie","family":"Acord","sequence":"additional","affiliation":[{"name":"Department of Neurology, Cook Children's Medical Center, Fort Worth, TX, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Warren","family":"Marks","sequence":"additional","affiliation":[{"name":"Department of Neurology, Cook Children's Medical Center, Fort Worth, TX, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2024,10,21]]},"reference":[{"key":"bibr1-08830738241284683","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01430-8"},{"key":"bibr2-08830738241284683","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000012910"},{"key":"bibr3-08830738241284683","doi-asserted-by":"publisher","DOI":"10.1017\/S1047951119002610"},{"key":"bibr4-08830738241284683","doi-asserted-by":"publisher","DOI":"10.1186\/1532-429X-15-107"},{"key":"bibr5-08830738241284683","doi-asserted-by":"publisher","DOI":"10.1016\/S0959-437X(02)00309-X"},{"key":"bibr6-08830738241284683","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002891"},{"key":"bibr7-08830738241284683","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2006.09.016"},{"key":"bibr8-08830738241284683","doi-asserted-by":"publisher","DOI":"10.3389\/fphys.2021.647010"},{"issue":"4","key":"bibr9-08830738241284683","first-page":"513","volume":"33","author":"Jacobs PA","year":"1981","journal-title":"Am J Hum Genet."},{"key":"bibr10-08830738241284683","doi-asserted-by":"publisher","DOI":"10.1111\/febs.15466"},{"key":"bibr11-08830738241284683","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2022.997551"},{"key":"bibr12-08830738241284683","doi-asserted-by":"publisher","DOI":"10.3390\/diagnostics11040701"},{"key":"bibr13-08830738241284683","doi-asserted-by":"publisher","DOI":"10.1002\/ana.24303"},{"key":"bibr14-08830738241284683","doi-asserted-by":"publisher","DOI":"10.18553\/jmcp.2020.26.4.361"},{"key":"bibr15-08830738241284683","volume-title":"FDA approves first gene therapy for treatment of certain patients with Duchenne muscular dystrophy","author":"Kempler C","year":"2023"},{"key":"bibr16-08830738241284683","volume-title":"Sarepta prices Duchenne gene therapy at $3.2M","author":"Fidler B","year":"2023"},{"key":"bibr17-08830738241284683","doi-asserted-by":"publisher","DOI":"10.1051\/medsci\/201733s104"},{"key":"bibr18-08830738241284683","unstructured":"Rubin M. 2023.\n                      Myotonia congenita - children\u2019s health issues\n                      . 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