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L

Louise R. Rodino-Klapac

sarepta therapeutics inc.
H指数:45
被引数:9,911

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Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial

杜氏肌营养不良症非卧床患者接受Delandistrogene Moxeparvovec治疗后的两年结果：EMBARK三期临床试验

Neurology and Therapy
IF4.8
2026-01-100
OAAI
Jerry R. Mendell; Francesco Muntoni; Craig M. McDonald; Eugenio M. Mercuri; Emma Ciafaloni; Hirofumi Komaki; Carmen Leon-Astudillo; Andrés Nascimento; Crystal Proud; Ulrike Schara-Schmidt; Aravindhan Veerapandiyan; Craig M. Zaidman; Matthew Furgerson; Kai Ding; Preeti Singh; Rachael Potter; Damon R. Asher; Alexander P. Murphy; Carol Reid; Gregory Hooper; Carmen O. Torre; Marianna Manfrini; Louise R. Rodino-Klapac
Pharmacokinetic and Pharmacodynamic Evaluation of Bidridistrogene Xeboparvovec in an Aged Murine Model of Limb-Girdle Muscular Dystrophy Type 2E/R4

Bidridistrogene Xeboparvovec 在 2E/R4 型 limb-girdle 肌营养不良老年小鼠模型中的药代动力学和药效动力学评估

Human Gene Therapy
IF4
2025-10-300
PRE
Stephen Baine; Jared Cui; Young-Eun Seo; Amber Kempton; Oliver Rogers; Alex Haile; Kaitlin Adegboye; Jasmine Wu; Luke Lemmerman; Mariana Guerrero; Caitlin Jones; Hannah Mayes; Tesla Freehafer; Sarah Lewis; Eric Pozsgai; Lilly East; Louise Rodino-Klapac; Rachael Potter
Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy An Exploratory Analysis From the EMBARK Randomized Clinical Trial

杜氏肌营养不良症患者定量肌肉磁共振结局：EMBARK随机临床试验的探索性分析

JAMA NEUROLOGY
IF21.3
2025-05-120
PRE
Vandenborne, Krista; Walter, Glenn A.; Straub, Volker; Willcocks, Rebecca J.; Forbes, Sean C.; Mercuri, Eugenio M.; Muntoni, Francesco; Ding, Kai; Ennamuri, Sravya; Reid, Carol; Murphy, Alexander P.; Manfrini, Marianna; Mendell, Jerry R.; Elkins, Jacob S.; Rodino-Klapac, Louise R.
Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy

delandistrogene moxeparvovec基因治疗后转基因定向免疫介导的肌炎的免疫学研究

SCIENTIFIC REPORTS
IF3.9
2025-01-021
OAAI
Potter, Rachael A.; Moeller, Ida H.; Khan, Sohrab; Haegel, Helene; Hollenstein, Andreas; Steiner, Guido; Wandel, Christoph; Murphy, Alexander P.; Asher, Damon R.; Palatinsky, Emanuel; Griffin, Danielle A.; Mason, Stefanie; Iannaccone, Susan T.; Zaidman, Craig M.; Rodino-Klapac, Louise R.
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

AAV基因治疗杜氏肌营养不良症: EMBARK 3期随机试验

NATURE MEDICINE
IF50
2024-10-093
OAAI
Mendell, Jerry R.; Muntoni, Francesco; McDonald, Craig M.; Mercuri, Eugenio M.; Ciafaloni, Emma; Komaki, Hirofumi; Leon-Astudillo, Carmen; Nascimento, Andres; Proud, Crystal; Schara-Schmidt, Ulrike; Veerapandiyan, Aravindhan; Zaidman, Craig M.; Guridi, Maitea; Murphy, Alexander P.; Reid, Carol; Wandel, Christoph; Asher, Damon R.; Darton, Eddie; Mason, Stefanie; Potter, Rachael A.; Singh, Teji; Zhang, Wenfei; Fontoura, Paulo; Elkins, Jacob S.; Rodino-Klapac, Louise R.
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
A phase 2 clinical trial evaluating the safety and efficacy of delandistrogene moxeparvovec for treating patients with Duchenne muscular dystrophy
GENETICS IN MEDICINE
IF6.2
2022-03-010
OA
O'Rourke, Erin; Mendell, Jerry; Shieh, Perry; Sahenk, Zarife; Lehman, Kelly; Lowes, Linda; Reash, Natalie; Iammarino, Megan; Alfano, Lindsay; Powers, Brenna; Woods, Jeremy; Skura, Christy; Mao, Howard; Staudt, Loretta; Potter, Rachael; Griffin, Danielle; Lewis, Sarah; Upadhyay, Sameer; Singh, Teji; Rodino-Klapac, Louise
Current Clinical Applications of In Vivo Gene Therapy with AAVs

AAVs体内基因治疗的临床应用现状

MOLECULAR THERAPY
IF12
2021-02-01452
OAAI
Mendell, Jerry R.; Al-Zaidy, Samiah A.; Rodino-Klapac, Louise R.; Goodspeed, Kimberly; Gray, Steven J.; Kay, Christine N.; Boye, Sanford L.; Boye, Shannon E.; George, Lindsey A.; Salabarria, Stephanie; Corti, Manuela; Byrne, Barry J.; Tremblay, Jacques P.

研究方向

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J
Jerry R. Mendell
H 指数:79论文数:310被引数:1.9万
Z
Zarife Sahenk
H 指数:56论文数:213被引数:1.1万
S
Sarah Lewis
H 指数:26论文数:84被引数:4,449
K
Kristin N. Heller
H 指数:15论文数:54被引数:990
R
Rachael A. Potter
H 指数:15论文数:48被引数:1,214
L
Lindsay N. Alfano
H 指数:32论文数:217被引数:7,717
L
Linda Lowes
H 指数:36论文数:272被引数:8,615
K
K. Reed Clark
H 指数:32论文数:79被引数:5,818
K
Kevin M. Flanigan
H 指数:68论文数:367被引数:1.5万
J
Jerry R. Mendell
H 指数:66论文数:290被引数:2.0万