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B

Barry J. Byrne

Catholic University of the Sacred Heart

80H指数
574论文数
2.6W被引数
收录论文 55
发表时间
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Thorough QT Study on the Effect of Therapeutic and Supratherapeutic Dosing of Givinostat in Healthy Volunteers吉文司他特治疗剂量和超治疗剂量对健康受试者QT间期影响的研究
errClinical Pharmacology in Drug Development
IF1.8
err2026-03-22
err0
PREAI
errMercuri, Eugenio; Byrne, Barry; Willis, Tracey; Bourke, John; Bettica, Paolo; Cazzaniga, Sara; Xue, Hongqi; Darpo, Borje
err分享
err收藏
A respiratory signature of disease progression in the Pompe rat庞贝病大鼠疾病进展的呼吸特征
errJournal of Neurophysiology
IF2.1
err2026-02-05
err0
PREAI
errMickle, Alyssa R.; Rana, Sabhya; Benevides, Ethan S.; Byrne, Barry J.; Fuller, David D.; Dale, Erica A.
err分享
err收藏
AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 TrialAAV9基因疗法在II型GM1神经节苷脂贮积症中的应用——一项I-II期临床试验
errNew England Journal of Medicine
IF78.5
err2026-02-01
err0
PREAI
errLewis, Connor J.; D'souza, Precilla; Johnston, Jean M.; Acosta, Maria T.; Farmer, Cristan; Baker, Eva H.; Crowell, Anna; Mojica, Yoliann; Ashraf, Sumaiya; Joseph, Lisa; Vezina, Gilbert; Quezado, Zenaide; Yousef, Muhammad H.; Vardar, Zeynep; Shazeeb, Mohammed Salman; Corti, Manuela; Blackwood, Meghan; Coleman, Kirsten; Batista, Rita; Thurm, Audrey; De Boever, Erika; Gahl, William A.; Byrne, Barry J.; Flotte, Terence R.; Jiang, Xuntian; Gross, Amanda L.; Keeler, Allison M.; Gray-Edwards, Heather; Martin, Douglas R.; Sena-Esteves, Miguel; Tifft, Cynthia J.
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Mapping glycogen accumulation and treatment effect in Pompe disease with saturation transfer MRI利用饱和转移MRI技术映射庞贝病中的糖原积累和治疗效应
errTranslational Research
IF5.9
err2026-02-01
err0
errOAAI
errZeng, Qing; Li, Yuguo; Timm, Derek; Johnson, Tyler; Mehta, Nickita; Martin, Lukas; Fox, Brian; van Zijl, Peter C. M.; Walter, Glenn A.; Rosenberg, Jens T.; Vander Kooi, Craig W.; Corti, Manuela; Gentry, Matthew S.; Sun, Ramon C.; Byrne, Barry J.; Yadav, Nirbhay N.
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Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe diseasemiglustat：一种用于治疗迟发性庞贝病的首创酶稳定剂，用于cipaglucosidase alfa
errTherapeutic Advances in Rare Disease
IF0
err2026-01-01
err0
PREAI
errHopkin, 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
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Cipaglucosidase alfa plus miglustat in Pompe disease: two non-ambulatory patients switching from high‑dose, high-frequency alglucosidase alfaCipaglucosidase alfa联合miglustat治疗庞贝病：2例从高剂量、高频率alglucosidase alfa转换治疗的无行走能力患者
errNeuromuscular Disorders
IF2.8
err2025-11-05
err0
PREAI
errBarry J. Byrne; Jeff Castelli; Vipul Jain; Sheela Sitaraman Das; Jennifer Zhang
err分享
err收藏
The future of gene therapy: Safer vectors, sharper focus基因治疗的前景：更安全的载体，更精准的靶向
errMolecular Therapy
IF12
err2025-09-12
err0
PREAI
errJeffrey S. Chamberlain; Beverly L. Davidson; Lindsey A. George; Barry J. Byrne; David Barrett
err分享
err收藏
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy关于在杜氏肌营养不良症患者中实施基因治疗及监测的共识推荐与考量
errNeuromuscular Disorders
IF2.8
err2025-08-23
err0
PREAI
errJodi M. Wolff; Nora Capocci; Evrim Atas; Diana X. Bharucha-Goebel; John F. Brandsema; Russell J. Butterfield; Christina B. Chadwick; Manuela Corti; Thomas O. Crawford; Linda Cripe; John W. Day; Tina Duong; Mai K. ElMallah; Kevin M. Flanigan; Lindsey A. George; Natalie L. Goedeker; Erica Goude; Sharon Hesterlee; Brian Lin; Natalie K. Katz; Susan E. Matesanz; Craig McDonald; Elizabeth M. McNally; Claudia Mercado-Rodriguez; Deipanjan Nandi; Julie A. Parsons; Crystal Proud; Leigh Ramos-Platt; Angela Lek; Stephanie M. Salabarria; Eric Camino; Rachel Schrader; Erin Shea; Richard Shell; Perry B. Shieh; Jonathan H. Soslow; Jane B. Taylor; Aravindhan Veerapandiyan; Chet Villa; Michele L. Yang; Craig M. Zaidman; Carmen Leon-Astudillo; Barry J. Byrne
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Complement activation in a phase Ib study of fordadistrogene movaparvovec for Duchenne muscular dystrophy福特达西罗金-莫瓦帕罗韦克（fordadistrogene movaparvovec）在杜氏肌营养不良症（Duchenne muscular dystrophy）的 Ib 期研究中的补体激活
errMolecular Therapy
IF12
err2025-06-28
err0
PREAI
errBarry J. Byrne; Russell J. Butterfield; Perry B. Shieh; Edward C. Smith; Christoph Licht; Michael Binks; Sandra Casinghino; Marielle Delnomdedieu; Kodihalli C. Ravindra; Tara McDonnell; Kelly Ryan; Martin Schulz; Qi Shen; Heliang Shi; Madhu P. Sirivelu; Vishal S. Vaidya; Laurence Whiteley; Daniel I. Levy
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Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease新生儿的全身性基因疗法恢复了庞贝病大鼠模型的心肺功能
errMolecular Therapy
IF12
err2025-06-14
err0
errOAAI
errDavid D. Fuller; Sabhya Rana; Prajwal P. Thakre; Ethan S. Benevides; Megan K. Pope; Adrian G. Todd; Victoria N. Jensen; Lauren Vaught; Denise A. Cloutier; Roberto A. Ribas; Reece C. Larson; Matthew S. Gentry; Ramon C. Sun; Vijay Chandran; Manuela Corti; Darin J. Falk; Barry J. Byrne
err分享
err收藏
Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL将晚期庞贝病糖苷酶替代疗法从阿糖苷酶alfa切换为西帕糖苷酶alfa联合米格鲁斯坦：PROPEL研究的后效分析
errMUSCLE & NERVE
IF3.1
err2025-05-07
err0
errOAAI
errHani Kushlaf; Jordi Díaz-Manera; Drago Bratkovic; Barry J. Byrne; Kristl G. Claeys; Paula R. Clemens; Mazen M. Dimachkie; Priya S. Kishnani; Pascal Laforêt; Mark Roberts; Benedikt Schoser; Antonio Toscano; Jeff Castelli; Fred Holdbrook; Sheela Sitaraman Das; Mitchell Goldman; Tahseen Mozaffar; the PROPEL Study Group
err分享
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Quantification and comparison of anti-AAV9 and anti-AAVrh74 antibodies in plasma and human milk: Implications for AAV-based gene therapy candidacyAAV9和AAVrh74抗体的定量与比较：在血浆和母乳中的检测及其对AAV基因治疗候选性的意义
errJOURNAL OF NEUROMUSCULAR DISEASES
IF3.4
err2025-04-10
err0
PREAI
errCarmen Leon-Astudillo; Kirsten Coleman; Stephanie M Salabarria; Vivian Valcarce; Lauren Stewart Stafford; Joseph Larkin; Josef Neu; Manuela Corti; Barry J Byrne
err分享
err收藏
Cardiopulmonary exercise testing as an integrative approach to explore physiological limitations in Duchenne muscular dystrophy心肺运动试验作为一种整合性方法，用于探索杜氏肌营养不良症中的生理限制。
errJOURNAL OF NEUROMUSCULAR DISEASES
IF3.4
err2025-03-04
err0
PREAI
errBomma, Meghana; Lott, Donovan; Forbes, Sean; Shih, Renata; Coppola, John-Anthony; Christle, Jeffrey W.; Duong, Tina; Russo, Joseph; Pant, Aditi; Leon-Astudillo, Carmen; Berthy, Julie; Cousins, Christina; Corti, Manuela; Byrne, Barry; May, James; Xue, W.; Taivassalo, Tanja
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Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease (Volume 15, 1451512, 2024)Cipaglucosidase alfa 联合 Miglustat：将作用机制与迟发型庞贝病的临床结局相联系（卷 15，1451512，2024）
errFRONTIERS IN NEUROLOGY
IF2.8
err2025-01-03
err1
errOAAI
errByrne, Barry J.; Parenti, Giancarlo; Schoser, Benedikt; van der Ploeg, Ans T.; Do, Hung; Fox, Brian; Goldman, Mitchell; Johnson, Franklin K.; Kang, Jia; Mehta, Nickita; Mondick, John; Sheikh, M. Osman; Sitaraman Das, Sheela; Tuske, Steven; Brudvig, Jon; Weimer, Jill M.; Mozaffar, Tahseen
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Neurological glycogen storage diseases and emerging therapeutics
errNEUROTHERAPEUTICS
IF6.9
err2024-09-01
err0
errOAAI
errColpaert, Matthieu; Singh, Pankaj K.; Donohue, Katherine J.; Pires, Natacha T.; Fuller, David D.; Corti, Manuela; Byrne, Barry J.; Sun, Ramon C.; Kooi, Craig W. Vander; Gentry, Matthew S.
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Meeting Report: 2023 Muscular Dystrophy Association Summit on 'Safety and Challenges in Gene Therapy of Neuromuscular Diseases'
errJOURNAL OF NEUROMUSCULAR DISEASES
IF3.4
err2024-08-03
err1
errOAAI
errLek, Angela; Atas, Evrim; Lin, Brian; Hesterlee, Sharon E.; Abbott, Jordan K.; Byrne, Barry J.; Bonnemann, Carsten G.
err分享
err收藏
In memoriam: Kenneth I. Berns, MD, PhD (1938-2024)
errMOLECULAR THERAPY
IF12
err2024-04-01
err0
errOAAI
errByrne, Barry J.; Flotte, Terence R.; Herzog, Roland W.; Srivastava, Arun
err分享
err收藏
NEO1/NEO-EXT studies: Long-term muscle quantitative magnetic resonance imaging and functional efficacy in adults with lateonset Pompe disease (LOPD) on avalglucosidase alfa treatment
errMOLECULAR GENETICS AND METABOLISM
IF3.5
err2024-02-01
err0
PREAI
errByrne, Barry; Carlier, Pierre G.; Vissing, John; Dimachkie, Mazen M.; Barohn, Richard; Kishnani, Priya S.; Ladha, Shafeeq; Mengelh, Eugen; Sacconi, Sabrina; Trivedi, Jaya; Young, Peter; Haack, Kristina An; Armstrong, Nicole; Miossec, Patrick; Thibault, Nathan; Sparks, Susan; Schoser, Benedikt; Diaz-Manera, Jordi
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err收藏
Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL
errMOLECULAR GENETICS AND METABOLISM
IF3.5
err2024-02-01
err1
PREAI
errMozaffar, Tahseen; Bratkovic, Drago; Byrne, Barry J.; Claeys, Kristl G.; Diaz-Manera, Jordi; Dimachkie, Mazen M.; Kishnani, Priya S.; Kushlaf, Hani; Roberts, Mark E.; Toscano, Antonio; Castelli, Jeffrey; Holdbrook, Fred K.; Das, Sheela Sitaraman; Wasfi, Yasmine; Schoser, Benedikt
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The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular DystrophyTaldefgrobep Alfa的临床开发: 一种抗肌生成抑制素Adnectin，用于治疗杜氏肌营养不良症
errNEUROLOGY AND THERAPY
IF4.8
err2024-01-08
err6
errOAAI
errMuntoni, Francesco; Byrne, Barry J.; Mcmillan, Hugh J.; Ryan, Monique M.; Wong, Brenda L.; Dukart, Juergen; Bansal, Amita; Cosson, Valerie; Dreghici, Roxana; Guridi, Maitea; Rabbia, Michael; Staunton, Hannah; Tirucherai, Giridhar S.; Yen, Karl; Yuan, Xiling; Wagner, Kathryn R.
err分享
err收藏

研究方向

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