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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR).
Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7.
Ann Neurol. 2023.
PMID: 37539981
Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy.
Goedeker NL, Dharia SD, Griffin DA, Coy J, Truesdale T, Parikh R, Whitehouse K, Santra S, Asher DR, Zaidman CM.
Goedeker NL, et al.
Ther Adv Neurol Disord. 2023 Jan 24;16:17562864221149781. doi: 10.1177/17562864221149781. eCollection 2023.
Ther Adv Neurol Disord. 2023.
PMID: 36710722
Free PMC article.
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Laboratory monitoring of nusinersen safety.
Goedeker NL, Gibbons JL, Varadhachary AS, Connolly AM, Zaidman CM.
Goedeker NL, et al.
Muscle Nerve. 2021 Jun;63(6):902-905. doi: 10.1002/mus.27217. Epub 2021 Mar 16.
Muscle Nerve. 2021.
PMID: 33675054
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Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series.
Chand DH, Zaidman C, Arya K, Millner R, Farrar MA, Mackie FE, Goedeker NL, Dharnidharka VR, Dandamudi R, Reyna SP.
Chand DH, et al. Among authors: goedeker nl.
J Pediatr. 2021 Apr;231:265-268. doi: 10.1016/j.jpeds.2020.11.054. Epub 2020 Nov 28.
J Pediatr. 2021.
PMID: 33259859
Free article.
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Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy.
Zaidman CM, Goedeker NL, Aqul AA, Butterfield RJ, Connolly AM, Crystal RG, Godwin KE, Hor KN, Mathews KD, Proud CM, Kula Smyth E, Veerapandiyan A, Watkins PB, Mendell JR.
Zaidman CM, et al. Among authors: goedeker nl.
J Neuromuscul Dis. 2024;11(3):687-699. doi: 10.3233/JND-230185.
J Neuromuscul Dis. 2024.
PMID: 38607761
Free PMC article.
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