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Three-Year Safety Results of SAR422459 (EIAV-ABCA4) Gene Therapy in Patients With ABCA4-Associated Stargardt Disease: An Open-Label Dose-Escalation Phase I/IIa Clinical Trial, Cohorts 1-5.
Am J Ophthalmol. 2022 Aug;240:285-301. doi: 10.1016/j.ajo.2022.02.013. Epub 2022 Mar 4.
Am J Ophthalmol. 2022.
PMID: 35248547
Free PMC article.
Clinical Trial.
Test-Retest Variability of Functional and Structural Parameters in Patients with Stargardt Disease Participating in the SAR422459 Gene Therapy Trial.
Parker MA, Choi D, Erker LR, Pennesi ME, Yang P, Chegarnov EN, Steinkamp PN, Schlechter CL, Dhaenens CM, Mohand-Said S, Audo I, Sahel J, Weleber RG, Wilson DJ.
Parker MA, et al. Among authors: chegarnov en.
Transl Vis Sci Technol. 2016 Oct 1;5(5):10. doi: 10.1167/tvst.5.5.10. eCollection 2016 Oct.
Transl Vis Sci Technol. 2016.
PMID: 27730010
Free PMC article.
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Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy.
Pennesi ME, Weleber RG, Yang P, Whitebirch C, Thean B, Flotte TR, Humphries M, Chegarnov E, Beasley KN, Stout JT, Chulay JD.
Pennesi ME, et al.
Hum Gene Ther. 2018 Dec;29(12):1428-1437. doi: 10.1089/hum.2018.014. Epub 2018 Jul 24.
Hum Gene Ther. 2018.
PMID: 29869534
Clinical Trial.
Item in Clipboard
Effect of Oral Valproic Acid vs Placebo for Vision Loss in Patients With Autosomal Dominant Retinitis Pigmentosa: A Randomized Phase 2 Multicenter Placebo-Controlled Clinical Trial.
Birch DG, Bernstein PS, Iannacone A, Pennesi ME, Lam BL, Heckenlively J, Csaky K, Hartnett ME, Winthrop KL, Jayasundera T, Hughbanks-Wheaton DK, Warner J, Yang P, Fish GE, Teske MP, Sklaver NL, Erker L, Chegarnov E, Smith T, Wahle A, VanVeldhuisen PC, McCormack J, Lindblad R, Bramer S, Rose S, Zilliox P, Francis PJ, Weleber RG.
Birch DG, et al.
JAMA Ophthalmol. 2018 Aug 1;136(8):849-856. doi: 10.1001/jamaophthalmol.2018.1171.
JAMA Ophthalmol. 2018.
PMID: 29879277
Free PMC article.
Clinical Trial.
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VFMA: Topographic Analysis of Sensitivity Data From Full-Field Static Perimetry.
Weleber RG, Smith TB, Peters D, Chegarnov EN, Gillespie SP, Francis PJ, Gardiner SK, Paetzold J, Dietzsch J, Schiefer U, Johnson CA.
Weleber RG, et al. Among authors: chegarnov en.
Transl Vis Sci Technol. 2015 Apr 28;4(2):14. doi: 10.1167/tvst.4.2.14. eCollection 2015 Apr.
Transl Vis Sci Technol. 2015.
PMID: 25938002
Free PMC article.
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