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Page 1
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.
Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR. Mendell JR, et al. Among authors: leon astudillo c. Nat Med. 2024 Oct 9. doi: 10.1038/s41591-024-03304-z. Online ahead of print. Nat Med. 2024. PMID: 39385046
Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies.
Salabarria SM, Corti M, Coleman KE, Wichman MB, Berthy JA, D'Souza P, Tifft CJ, Herzog RW, Elder ME, Shoemaker LR, Leon-Astudillo C, Tavakkoli F, Kirn DH, Schwartz JD, Byrne BJ. Salabarria SM, et al. Among authors: leon astudillo c. J Clin Invest. 2024 Jan 2;134(1):e173510. doi: 10.1172/JCI173510. J Clin Invest. 2024. PMID: 37988172 Free PMC article.
16 results