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Immune-mediated myositis following gene therapy for Duchenne muscular dystrophy: a case report

22 Jun 2024

Abstract excerpt

Clinical data were from the authors' observations and the electronic medical records of Children’s Health. Pre-treatment histologic samples were provided by Sarepta Therapeutics. The muscle biopsy was processed at the University of Texas Southwestern Medical Center. A 9-year-old ambulatory male patient, diagnosed with DMD at 2.5 years of age presented with acute-onset bulbar and limb girdle weakness 1 month...

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Share to Inflammatory Myopathies and Dermatomyositis
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PMID: 38907024
DOI: 10.1007/s00415-024-12431-z

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