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

Mapping glycogen accumulation and treatment effect in Pompe disease with saturation transfer MRI

利用饱和转移磁共振成像技术绘制庞贝病中糖原积累及治疗效果图

Zeng, Qing; Li, Yuguo; Timm, Derek; Johnson, Tyler; Mehta, Nickita; Martin, Lukas; Fox, Brian; van Zijl, Peter C M; Walter, Glenn A; Rosenberg, Jens T; Kooi, Craig W Vander; Corti, Manuela; Gentry, Matthew S; Sun, Ramon C; Byrne, Barry J; Yadav, Nirbhay N

Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series

研究总结:脊髓性肌萎缩症患者在接受奥那西姆诺基因阿贝帕沃韦治疗后接受利司迪普拉姆治疗:一项多中心病例系列研究

Svoboda, Melissa D; Kuntz, Nancy; Leon-Astudillo, Carmen; Byrne, Barry J; Krueger, Jena; Kwon, Jennifer M; Sieburg, Cory; Castro, Diana

A respiratory signature of disease progression in the Pompe rat

庞贝氏症大鼠疾病进展的呼吸特征

Mickle, Alyssa R; Rana, Sabhya; Benevides, Ethan S; Byrne, Barry J; Fuller, David D; Dale, Erica A

Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials

居家使用西帕葡萄糖苷酶α联合米格鲁司他治疗晚发型庞贝病的安全性:多项临床试验的结果

Andersen, Henning; Díaz-Manera, Jordi; Goker-Alpan, Ozlem; Mozaffar, Tahseen; Sitaraman Das, Sheela; Fox, Brian; Amon, Farah; O'Brien-Prince, Kinesha; Goldman, Mitchell; Holdbrook, Fred; Jain, Vipul; Byrne, Barry J

Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease

米格鲁司他:一种用于治疗晚发型庞贝病的西帕葡萄糖苷酶α的首创酶稳定剂

Hopkin, Robert J; Byrne, Barry J; Dimachkie, Mazen M; Kishnani, Priya S; Mozaffar, Tahseen; Roberts, Mark; Schoser, Benedikt; van der Beek, Nadine A M E; van der Ploeg, Ans T; Wenninger, Stephan; Brudvig, Jon; Fox, Brian; Holdbrook, Fred; Jain, Vipul; Johnson, Franklin; Zhang, Jennifer; Parenti, Giancarlo

Overexpression of Wild-Type TMEM43 Improves Cardiac Function in Arrhythmogenic Right Ventricular Cardiomyopathy Type 5.

野生型 TMEM43 的过表达可改善致心律失常性右室心肌病 5 型的心脏功能

Lalaguna Laura, Arévalo-Núñez de Arenas María, López-Olañeta Marina, Villalba-Orero María, Jiménez-Riobóo Rafael J, Gómez-Gaviro María Victoria, Isern Joan, Muñoz-Cánoves Pura, Byrne Barry J, Ochoa Juan Pablo, García-Pavía Pablo, Lara-Pezzi Enrique

The future of gene therapy: Safer vectors, sharper focus: High-profile failures demand deep root cause analysis - but the transformative potential of AAV remains within reach if the field is willing to learn and evolve

基因疗法的未来:更安全的载体,更精准的靶点:备受瞩目的失败案例需要进行深入的根本原因分析——但如果该领域愿意学习和发展,AAV的变革潜力仍然触手可及。

Chamberlain, Jeffrey S; Davidson, Beverly L; George, Lindsey A; Byrne, Barry J; Barrett, David

Clinical advances in gene, cell, and RNA therapies

基因、细胞和RNA疗法的临床进展

Milone, Michael C; Mansilla-Soto, Jorge; Pardi, Norbert; George, Lindsey A; Byrne, Barry J; Herzog, Roland W

Current clinical applications of AAV-mediated gene therapy

目前AAV介导的基因治疗的临床应用

Byrne, Barry J; Flanigan, Kevin M; Matesanz, Susan E; Finkel, Richard S; Waldrop, Megan A; D'Ambrosio, Eleonora S; Johnson, Nicholas E; Smith, Barbara K; Bönnemann, Carsten; Carrig, Sean; Rossano, Joseph W; Greenberg, Barry; Lalaguna, Laura; Lara-Pezzi, Enrique; Subramony, Sub; Corti, Manuela; Mercado-Rodriguez, Claudia; Leon-Astudillo, Carmen; Ahrens-Nicklas, Rebecca; Bharucha-Goebel, Diana; Gao, Guangping; Gessler, Dominic J; Hwu, Wuh-Liang; Chien, Yin-Hsiu; Lee, Ni-Chung; Boye, Sanford L; Boye, Shannon E; George, Lindsey A

Complement activation in a phase Ib study of fordadistrogene movaparvovec for Duchenne muscular dystrophy

在针对杜氏肌营养不良症的 fordadistrogene movaparvovec 的 Ib 期研究中,补体激活

Byrne, Barry J; Butterfield, Russell J; Shieh, Perry B; Smith, Edward C; Licht, Christoph; Binks, Michael; Casinghino, Sandra; Delnomdedieu, Marielle; Ravindra, Kodihalli C; McDonnell, Tara; Ryan, Kelly; Schulz, Martin; Shen, Qi; Shi, Heliang; Sirivelu, Madhu P; Vaidya, Vishal S; Whiteley, Laurence; Levy, Daniel I