日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

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

SAR422459 (EIAV-ABCA4) 基因疗法治疗 ABCA4 相关 Stargardt 病患者的三年安全性结果:一项开放标签剂量递增 I/IIa 期临床试验,队列 1-5

Parker, Maria A; Erker, Laura R; Audo, Isabelle; Choi, Dongseok; Mohand-Said, Saddek; Sestakauskas, Kastytis; Benoit, Patrick; Appelqvist, Terence; Krahmer, Melissa; Ségaut-Prévost, Caroline; Lujan, Brandon J; Faridi, Ambar; Chegarnov, Elvira N; Steinkamp, Peter N; Ku, Cristy; da Palma, Mariana Matioli; Barale, Pierre-Olivier; Ayelo-Scheer, Sarah; Lauer, Andreas; Stout, Tim; Wilson, David J; Weleber, Richard G; Pennesi, Mark E; Sahel, José Alain; Yang, Paul

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

口服丙戊酸与安慰剂治疗常染色体显性遗传性视网膜色素变性患者视力丧失的效果:一项随机、多中心、安慰剂对照的II期临床试验

Birch, David G; Bernstein, Paul S; Iannacone, Alessandro; Pennesi, Mark E; Lam, Byron L; Heckenlively, John; Csaky, Karl; Hartnett, Mary Elizabeth; Winthrop, Kevin L; Jayasundera, Thiran; Hughbanks-Wheaton, Dianna K; Warner, Judith; Yang, Paul; Fish, Gary Edd; Teske, Michael P; Sklaver, Neal L; Erker, Laura; Chegarnov, Elvira; Smith, Travis; Wahle, Aimee; VanVeldhuisen, Paul C; McCormack, Jennifer; Lindblad, Robert; Bramer, Steven; Rose, Stephen; Zilliox, Patricia; Francis, Peter J; Weleber, Richard G

Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy

RPE65缺陷型视网膜营养不良基因治疗5年后的结果

Pennesi, Mark E; Weleber, Richard G; Yang, Paul; Whitebirch, Chris; Thean, Beverly; Flotte, Terence R; Humphries, Margaret; Chegarnov, Elvira; Beasley, Kathleen N; Stout, J Timothy; Chulay, Jeffrey D

Test-Retest Variability of Functional and Structural Parameters in Patients with Stargardt Disease Participating in the SAR422459 Gene Therapy Trial

参与 SAR422459 基因治疗试验的 Stargardt 病患者功能和结构参数的重测变异性

Parker, Maria A; Choi, Dongseok; Erker, Laura R; Pennesi, Mark E; Yang, Paul; Chegarnov, Elvira N; Steinkamp, Peter N; Schlechter, Catherine L; Dhaenens, Claire-Marie; Mohand-Said, Saddek; Audo, Isabelle; Sahel, Jose; Weleber, Richard G; Wilson, David J

VFMA: Topographic Analysis of Sensitivity Data From Full-Field Static Perimetry

VFMA:全视野静态视野敏感度数据的地形分析

Weleber, Richard G; Smith, Travis B; Peters, Dawn; Chegarnov, Elvira N; Gillespie, Scott P; Francis, Peter J; Gardiner, Stuart K; Paetzold, Jens; Dietzsch, Janko; Schiefer, Ulrich; Johnson, Chris A