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Phase I study of liver depot gene therapy in late-onset Pompe disease.
Smith EC, Hopkins S, Case LE, Xu M, Walters C, Dearmey S, Han SO, Spears TG, Chichester JA, Bossen EH, Hornik CP, Cohen JL, Bali D, Kishnani PS, Koeberl DD. Smith EC, et al. Mol Ther. 2023 Jul 5;31(7):1994-2004. doi: 10.1016/j.ymthe.2023.02.014. Epub 2023 Feb 18. Mol Ther. 2023. PMID: 36805083 Clinical Trial.
Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.
Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, McDonald CM, Smith EC, Zaidman CM, Nakagawa T; CINRG DNHS Investigators; Hoffman EP. Clemens PR, et al. Among authors: smith ec. J Neuromuscul Dis. 2023;10(3):439-447. doi: 10.3233/JND-221656. J Neuromuscul Dis. 2023. PMID: 37005891 Free PMC article. Clinical Trial.
The severity of MUSK pathogenic variants is predicted by the protein domain they disrupt.
Cocanougher BT, Liu SW, Francescatto L, Behura A, Anneling M, Jackson DG, Deak KL, Hornik CD, ElMallah MK, Pizoli CE, Smith EC, Tan KGQ, McDonald MT. Cocanougher BT, et al. Among authors: smith ec. HGG Adv. 2024 Apr 1;5(3):100288. doi: 10.1016/j.xhgg.2024.100288. Online ahead of print. HGG Adv. 2024. PMID: 38566418 Free PMC article.
Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy.
Peay HL, Fischer R, Mange B, Paquin RS, Smith EC, Sadosky A, Russo L, Ricotti V, Rensch C, Morris C, Martin AS, Ganot A, Beaverson K, Mansfield C. Peay HL, et al. Among authors: smith ec. Mol Genet Genomic Med. 2021 May;9(5):e1664. doi: 10.1002/mgg3.1664. Epub 2021 Mar 23. Mol Genet Genomic Med. 2021. PMID: 33755338 Free PMC article.
395 results