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Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy.
Potter RA, Moeller IH, Khan S, Haegel H, Hollenstein A, Steiner G, Wandel C, Murphy AP, Asher DR, Palatinsky E, Griffin DA, Mason S, Iannaccone ST, Zaidman CM, Rodino-Klapac LR. Potter RA, et al. Among authors: iannaccone st. Sci Rep. 2025 Jan 2;15(1):4. doi: 10.1038/s41598-024-84077-w. Sci Rep. 2025. PMID: 39747998 Free PMC article. Clinical Trial.
Ectopic PLAG1 induces muscular dystrophy in the mouse.
Aguayo JS, Shelton JM, Tan W, Rakheja D, Cai C, Shalaby A, Lee J, Iannaccone ST, Xu L, Chen K, Burns DK, Zheng Y. Aguayo JS, et al. Among authors: iannaccone st. Biochem Biophys Res Commun. 2023 Jul 12;665:159-168. doi: 10.1016/j.bbrc.2023.05.006. Epub 2023 May 3. Biochem Biophys Res Commun. 2023. PMID: 37163936
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG).
Finkel RS, Darras BT, Mendell JR, Day JW, Kuntz NL, Connolly AM, Zaidman CM, Crawford TO, Butterfield RJ, Shieh PB, Tennekoon G, Brandsema JF, Iannaccone ST, Shoffner J, Kavanagh S, Macek TA, Tauscher-Wisniewski S. Finkel RS, et al. Among authors: iannaccone st. J Neuromuscul Dis. 2023;10(3):389-404. doi: 10.3233/JND-221560. J Neuromuscul Dis. 2023. PMID: 36911944 Free PMC article.
New results for risdiplam in spinal muscular atrophy.
Iannaccone ST. Iannaccone ST. Lancet Neurol. 2022 Dec;21(12):1065-1066. doi: 10.1016/S1474-4422(22)00427-6. Epub 2022 Oct 14. Lancet Neurol. 2022. PMID: 36244363 No abstract available.
163 results