Immune-mediated myositis following gene therapy for Duchenne muscular dystrophy: a case report
Author:
Funder
Sarepta Therapeutics
Publisher
Springer Science and Business Media LLC
Link
https://link.springer.com/content/pdf/10.1007/s00415-024-12431-z.pdf
Reference12 articles.
1. Matthews E, Brassington R, Kuntzer T, Jichi F, Manzur AY (2016) Corticosteroids for the treatment of Duchenne muscular dystrophy. Cochrane Database Syst Rev 2016:CD003725. https://doi.org/10.1002/14651858.CD003725.pub4
2. Takeda S, Clemens PR, Hoffman EP (2021) Exon-skipping in Duchenne muscular dystrophy. J Neuromuscul Dis 8:S343–S358. https://doi.org/10.3233/JND-210682
3. ELEVIDYSTM Highlights of prescribing information. US Food and Drug Administration; 2023.
4. Mendell JR, Rodino-Klapac L, Sahenk Z, Malik V, Kaspar BK, Walker CM, Clark KR (2012) Gene therapy for muscular dystrophy: lessons learned and path forward. Neurosci Lett 527:90–99. https://doi.org/10.1016/j.neulet.2012.04.078
5. Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR (2020) Assessment of systemic delivery of rAAVrh74.MHCK7.micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial. JAMA Neurol 77:1122–1131. https://doi.org/10.1001/jamaneurol.2020.1484
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