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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM. Russell S, et al. Among authors: davis m. Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14. Lancet. 2017. PMID: 28712537 Free PMC article. Clinical Trial.
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.
Maguire AM, Russell S, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, Marshall KA, McCague S, Reichert H, Davis M, Simonelli F, Leroy BP, Wright JF, High KA, Bennett J. Maguire AM, et al. Among authors: davis m. Ophthalmology. 2019 Sep;126(9):1273-1285. doi: 10.1016/j.ophtha.2019.06.017. Epub 2019 Jun 22. Ophthalmology. 2019. PMID: 31443789 Free article. Clinical Trial.
A high dose KRP203 induces cytoplasmic vacuoles associated with altered phosphoinositide segregation and endosome expansion.
Kofuji S, Wolfe K, Sumita K, Kageyama S, Yoshino H, Hirota Y, Ogawa-Iio A, Kanoh H, Sasaki M, Kofuji K, Davis MI, Pragani R, Shen M, Boxer MB, Nakatsu F, Nigorikawa K, Sasaki T, Takeuchi K, Senda T, Kim SM, Edinger AL, Simeonov A, Sasaki AT. Kofuji S, et al. Among authors: davis mi. Biochem Biophys Res Commun. 2024 Apr 27;718:149981. doi: 10.1016/j.bbrc.2024.149981. Online ahead of print. Biochem Biophys Res Commun. 2024. PMID: 38735134
Response to letter.
Amin KS, Forman HP, Davis MA. Amin KS, et al. Among authors: davis ma. Clin Imaging. 2024 May 7;111:110173. doi: 10.1016/j.clinimag.2024.110173. Online ahead of print. Clin Imaging. 2024. PMID: 38735100 No abstract available.
9,597 results