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P

Perry B. Shieh

university of california at los angeles
H指数:48
被引数:1.0万

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教育背景

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工作经历

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荣誉奖项

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Establishing biomarkers and clinical endpoints in myotonic dystrophy type 1 (END-DM1): Protocol of an international natural history study

在强直性肌营养不良1型中建立生物标志物和临床终点(END-DM1)：一项国际性自然病史研究的方案

PLOS ONE
IF2.6
2025-12-110
PRE
Mul, Karlien; Eichinger, Kate; Hung, Man; Sansone, Valeria A.; Gagnon, Cynthia; Subramony, Sub; Roxburgh, Richard H.; Hamel, Johanna; Statland, Jeffrey M.; Elsheikh, Bakri; Turner, Chris; Sampson, Jacinda; Ragole, Thomas; Matthews, Emma; Schoser, Benedikt; Swenson, Andrea; Laverty, Chamindra; Shieh, Perry; Greene, Ericka P.; Takahashi, Masanori; Wicklund, Matthew; Dekdebrun, Jeanne; Raymond, Jennifer; Despain, Erin; Thornton, Charles A.; Johnson, Nicholas E.
Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry

气管切开与治疗：RESTORE登记研究中，接受onasemnogene abeparvovec治疗的气管切开术患者的脊髓性肌萎缩症患者的安全性与运动结局

Journal of Neuromuscular Diseases
IF3.4
2025-11-110
OA
Yasemin Erbaş; Laurent Servais; Perry B Shieh; Natalie L Goedeker; Megan A Waldrop; Ryosuke Bo; Dheeraj Raju; Kamal Benguerba; Sandra P Reyna; David Wolff; Richard S Finkel
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

Neuromuscular Disorders
IF2.8
2025-08-230
PRE
Jodi 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
Switching to subcutaneous zilucoplan from intravenous complement component 5 inhibitors in generalised myasthenia gravis: a phase IIIb, open-label study

从静脉补体成分5抑制剂转换为皮下注射zilucoplan治疗重症肌无力：一项IIIb期、开放标签研究

Therapeutic Advances in Neurological Disorders
IF4.1
2025-07-050
OA
Miriam Freimer; Urvi Desai; Raghav Govindarajan; Min K. Kang; Shaida Khan; Bhupendra Khatri; Todd Levine; Samir Macwan; Perry B. Shieh; Michael D. Weiss; Jos Bloemers; Babak Boroojerdi; Eumorphia Maria Delicha; Andreea Lavrov; Puneet Singh; James F. Howard
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

Molecular Therapy
IF12
2025-06-280
PRE
Barry 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
Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchenne muscular dystrophy: Initial observations from a phase 1b trial

福特达斯特罗金莫瓦帕罗韦基（fordadistrogene movaparvovec）基因疗法在杜氏肌营养不良症中的心脏安全性：1b期试验的初步观察

Molecular Therapy
IF12
2025-06-280
PREAI
Sarah P. Sherlock; Daniel I. Levy; Avery McIntosh; Perry B. Shieh; Edward C. Smith; Tara G. McDonnell; Kelly A. Ryan; Marielle Delnomdedieu; Michael Binks; Ashwin K. Lal; Russell J. Butterfield
Strength and functional correlates of reachable workspace in facioscapulohumeral muscular dystrophy

面肩肱型肌营养不良症中可达工作空间的力量和功能相关性

NEUROMUSCULAR DISORDERS
IF2.8
2025-03-010
PRE
Wang, Leo H.; Hatch, Maya N.; Mcdermott, Michael P.; Martens, William B.; Eichinger, Katy; Lewis, Leann; Walker, Michaela; Leung, Doris G.; Wagner, Kathryn R.; Sacconi, Sabrina; Mul, Karlien; Shieh, Perry B.; Elsheikh, Bakri; Butterfield, Russell J.; Johnson, Nicholas E.; Sansone, Valeria; Han, Jay J.; Tawil, Rabi; Statland, Jeffrey M.; ReSolve Investigators FSHD CTRN
Current insights in ultra-rare adenylosuccinate synthetase 1 myopathy - meeting report on the First Clinical and Scientific Conference. 3 June 2024, National Centre for Advancing Translational Science, Rockville, Maryland, the United States of America
ORPHANET JOURNAL OF RARE DISEASES
IF3.5
2024-11-261
OAAI
Rybalka, Emma; Park, Hyung Jun; Nalini, Atchayaram; Baskar, Dipti; Polavarapu, Kiran; Durmus, Hacer; Xia, Yang; Wan, Linlin; Shieh, Perry B.; Moghadaszadeh, Behzad; Beggs, Alan H.; Mack, David L.; Smith, Alec S. T.; Hanna-Rose, Wendy; Jinnah, Hyder A.; Timpani, Cara A.; Shen, Min; Upadhyay, Jaymin; Brault, Jeffrey J.; Hall, Matthew D.; Baweja, Naveen; Kakkar, Priyanka
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy

Vamorolone在Duchenne肌营养不良症男孩中48周的疗效和安全性

NEUROLOGY
IF8.5
2024-03-1211
OAAI
Dang, Utkarsh J.; Damsker, Jesse M.; Guglieri, Michela; Clemens, Paula R.; Perlman, Seth J.; Smith, Edward C.; Horrocks, Iain; Finkel, Richard S.; Mah, Jean K.; Deconinck, Nicolas; Goemans, Nathalie M.; Haberlova, Jana; Straub, Volker; Mengle-Gaw, Laurel; Schwartz, Benjamin D.; Harper, Amy; Shieh, Perry B.; De Waele, Liesbeth; Castro, Diana; Yang, Michele L.; Ryan, Monique M.; McDonald, Craig M.; Tulinius, Mar; Webster, Richard I.; Mcmillan, Hugh J.; Kuntz, Nancy; Rao, Vamshi K.; Baranello, Giovanni; Spinty, Stefan; Childs, Anne-Marie; Sbrocchi, Annie M.; Selby, Kathryn A.; Monduy, Migvis; Nevo, Yoram; Vilchez, Juan J.; Nascimento-Osorio, Andres; Niks, Erik H.; De Groot, Imelda J. M.; Katsalouli, Marina; van den Anker, John N.; Ward, Leanne M.; Leinonen, Mika; D'Alessandro, Andrea L.; Hoffman, Eric P.
Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

用Onasemnogene Abeparvovec单药治疗的脊髓性肌萎缩症患者的真实世界结果: 来自RESTORE注册中心的发现

JOURNAL OF NEUROMUSCULAR DISEASES
IF3.4
2024-01-1820
OAAI
Servais, Laurent; Day, John W.; De Vivo, Darryl C.; Kirschner, Janbernd; Mercuri, Eugenio; Muntoni, Francesco; Proud, Crystal M.; Shieh, Perry B.; Tizzano, Eduardo F.; Quijano-Roy, Susana; Desguerre, Isabelle; Saito, Kayoko; Faulkner, Eric; Benguerba, Kamal M.; Raju, Dheeraj; LaMarca, Nicole; Sun, Rui; Anderson, Frederick A.; Finkel, Richard S.
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial
EBIOMEDICINE
IF10.8
2024-01-010
OAAI
Lawlor, Michael W.; Schoser, Benedikt; Margeta, Marta; Sewry, Caroline A.; Jones, Karra A.; Shieh, Perry B.; Kuntz, Nancy L.; Smith, Barbara K.; Dowling, James J.; Mueller-Felber, Wolfgang; Boennemann, Carsten G.; Seferian, Andreea M.; Blaschek, Astrid; Neuhaus, Sarah; Foley, A. Reghan; Saade, Dimah N.; Tsuchiya, Etsuko; Qasim, Ummulwara R.; Beatka, Margaret; Prom, Mariah J.; Ott, Emily; Danielson, Susan; Krakau, Paul; Kumar, Suresh N.; Meng, Hui; Vanden Avond, Mark; Wells, Clive; Gordish-Dressman, Heather; Beggs, Alan H.; Christensen, Sarah; Conner, Edward; James, Emma S.; Lee, Jun; Sadhu, Chanchal; Miller, Weston; Sepulveda, Bryan; Varfaj, Fatbardha; Prasad, Suyash; Rico, Salvador
Recurrent de novo SPTLC2 variant causes childhood-onset amyotrophic lateral sclerosis (ALS) by excess sphingolipid synthesis
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY
IF7.5
2023-11-246
OAAI
Syeda, Safoora B.; Lone, Museer A.; Mohassel, Payam; Donkervoort, Sandra; Munot, Pinki; Franca, Marcondes C.; Galarza-Brito, Juan Eli; Eckenweiler, Matthias; Asamoah, Alexander; Gable, Kenneth; Majumdar, Anirban; Schumann, Anke; Gupta, Sita D.; Lakhotia, Arpita; Shieh, Perry B.; Foley, A. Reghan; Jackson, Kelly E.; Chao, Katherine R.; Winder, Thomas L.; Catapano, Francesco; Feng, Lucy; Kirschner, Janbernd; Muntoni, Francesco; Dunn, Teresa M.; Hornemann, Thorsten; Bonnemann, Carsten G.
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

鞘内注射Onasemnogene Abeparvovec用于坐着，非卧床的脊髓性肌萎缩症患者: I期递增剂量研究 (STRONG)

JOURNAL OF NEUROMUSCULAR DISEASES
IF3.4
2023-05-0221
OAAI
Finkel, Richard S.; Darras, Basil T.; Mendell, Jerry R.; Day, John W.; Kuntz, Nancy L.; Connolly, Anne M.; Zaidman, Craig M.; Crawford, Thomas O.; Butterfield, Russell J.; Shieh, Perry B.; Tennekoon, Gihan; Brandsema, John F.; Iannaccone, Susan T.; Shoffner, John; Kavanagh, Sarah; Macek, Thomas A.; Tauscher-Wisniewski, Sitra
The FSHD Composite Outcome Measure (FSHD-COM) is Reliable, Valid, and Measures Disease Progression
NEUROLOGY
IF8.5
2023-04-250
PRE
Eichinger, Katy; McDermott, Michael; Higgs, Kiley; Walker, Michaela; Lewis, Leann; Martens, William; Leung, Doris; Stinson, Nikia; Holzer, Megan M.; Sacconi, Sabrina; Garcia, Jeremy; Benito, Victor De Paz; Mul, Karlien; Sansone, Valeria; Carraro, Elena; Becchiati, Stefano; Frisoni, Maria Chiara; Wang, Leo; Kieu, Catherine; Shieh, Perry; Skura, Christy; Elsheikh, Bakri; Kelly, Kristina; Jaworek, Andrea; LoRusso, Samantha; Butterfield, Russell; Wilson, Amelia; McIntyre, Melissa; Johnson, Nicholas; Butler, Amanda; Jones, Aileen; Hayes, Melissa; Baker, Lindsay; Sasidharan, Sandhya; Tawil, Alrabi; Statland, Jeffrey
Motor Outcomes to Validate Evaluations in Facioscapulohumeral muscular dystrophy (MOVE FSHD): Preliminary Baseline Characteristics

验证面肩肱型肌营养不良症 (MOVE FSHD) 评估的运动结果: 初步基线特征

NEUROLOGY
IF8.5
2023-04-250
PRE
Walker, Michaela; Butterfield, Russell; Day, John; Eichinger, Katy; Elsheikh, Bakri; Friedman, Seth; Genge, Angela; Higgs, Kiley; Johnson, Nicholas; Jones, Peter; Leung, Doris; Lewis, Leann; Lochmuller, Hanns; O'Ferrall, Erin; Martens, William; Shaw, Dennis; Shieh, Perry; Subramony, S.; Trivedi, Jaya; Wang, Leo; Wicklund, Matthew; Tawil, Alrabi; Statland, Jeffrey
Integrated Analyses of Data from Clinical Trials of Delandistrogene Moxeparvovec (SRP-9001) in Duchenne Muscular Dystrophy (DMD)

Duchenne肌营养不良症 (DMD) 的dlandistrogene Moxeparvovec (SRP-9001) 临床试验数据的综合分析

NEUROLOGY
IF8.5
2023-04-250
PRE
Proud, Crystal; Zaidman, Craig; Shieh, Perry; McDonald, Craig; Day, John; Mason, Stefanie; Guridi, Maitea; Han, Lixin; Yu, Lixi; Reid, Carol; Darton, Eddie; Wandel, Christoph; Richardson, James; Malhotra, Jyoti; Singh, Tejdip; Rodino-Klapac, Louise; Mendell, Jerry
Quantitative Muscle Analysis in FSHD Using Whole-Body Fat-Referenced MRI Composite Scores for Longitudinal and Cross-sectional Analysis
NEUROLOGY
IF8.5
2022-08-3016
OA
Mellion, Michelle L.; Widholm, Per; Karlsson, Markus; Ahlgren, Andre; Tawil, Rabi; Wagner, Kathryn R.; Statland, Jeffrey M.; Wang, Leo; Shieh, Perry B.; van Engelen, Baziel G. M.; Kools, Joost; Ronco, Lucienne; Odueyungbo, Adefowope; Jiang, John; Han, Jay J.; Hatch, Maya; Towles, Jeanette; Leinhard, Olof Dahlqvist; Cadavid, Diego
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
NATURE MEDICINE
IF50
2022-06-17143
OAAI
Strauss, Kevin A.; Farrar, Michelle A.; Muntoni, Francesco; Saito, Kayoko; Mendell, Jerry R.; Servais, Laurent; McMillan, Hugh J.; Finkel, Richard S.; Swoboda, Kathryn J.; Kwon, Jennifer M.; Zaidman, Craig M.; Chiriboga, Claudia A.; Iannaccone, Susan T.; Krueger, Jena M.; Parsons, Julie A.; Shieh, Perry B.; Kavanagh, Sarah; Wigderson, Melissa; Tauscher-Wisniewski, Sitra; McGill, Bryan E.; Macek, Thomas A.

研究方向

暂未获取到该数据
R
Richard S. Finkel
H 指数:83论文数:471被引数:3.1万
J
John Day
H 指数:94论文数:871被引数:3.5万
N
Nancy L. Kuntz
H 指数:49论文数:204被引数:1.3万
A
Anne M. Connolly
H 指数:66论文数:257被引数:1.7万
S
Susan T. Iannaccone
H 指数:62论文数:280被引数:1.7万
B
Basil T. Darras
H 指数:72论文数:517被引数:2.2万
R
Russell J. Butterfield
H 指数:33论文数:168被引数:4,154
L
Laurent Servais
H 指数:63论文数:603被引数:1.5万
E
Eugenio Mercuri
H 指数:108论文数:1,133被引数:4.8万
C
Claudia A. Chiriboga
H 指数:38论文数:148被引数:1.0万