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Kevin M. Flanigan

nationwide children's hospital and abigail wexner research institute

68H指数
365论文数
1.6W被引数
收录论文 105
发表时间
Plasma EV Proteomics Identifies ECM Remodeling and Inflammatory Proteins LUM and C7 as Candidate Biomarkers in FSHD血浆外泌体蛋白质组学鉴定出ECM重塑和炎症蛋白LUM和C7作为FMD的候选生物标志物
err2026-05-20
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errOAAI
errMustafa Bilal Bayazit; Chiranth K. Nagaraj; Jackson S. Newell; Kim Truc Nguyen; Xilal Y. Rima; Jacob Doon-Ralls; Eduardo Reátegui; Jeffrey M. Statland; Rabi Tawil; Kevin M. Flanigan; Scott Q. Harper; Nizar Y. Saad
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Unraveling the spatial landscape of dystrophinopathies: a transcriptomic approach to Becker and Duchenne muscular dystrophies揭开肌营养不良症的时空景观:一种针对贝克和杜氏肌营养不良症的转录组学方法
err2026-05-01
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errLaura GM Heezen; Qirong Mao; Stefan Nicolau; Claudio Novella Rausell; Julia van der Weerd; Jan Kueckelhaus; Rasya Gokul Nath; Jordi Diaz- Manera; Hermien E Kan; Erik H Niks; Maaike van Putten; Annemieke Aartsma-Rus; Kevin M Flanigan; Ahmed Mahfouz; Pietro Spitali
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Predicting subtypes of glycogen storage disease type IV: Challenges of hepatic subtypes and genotype-phenotype correlation预测糖原贮积症IV型的亚型:肝源性亚型的挑战及基因型-表型相关性
err2025-11-24
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PREAI
errAnne Taylor; Desale Yacob; Bonita Fung; Shamlal Mangray; Daniel R. Boué; Kevin M. Flanigan; Rebecca L. Koch; Priya S. Kishnani; Deeksha Bali; Alexander Weymann; Mari Mori
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Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK重新评估基因治疗的可接受死亡风险:一项在美国和英国杜氏肌营养不良症患者及其护理人员中的阈值研究
err2025-10-01
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errOAAI
errPeay, Holly; Fischer, Ryan; McNiff, Megan; Heslop, Emma; Pierce, Anna; Denger, Brian; Camino, Eric; Johnson, Alexandra; Cope, Heidi; Hill, Christine; Beaverson, Katherine L.; Ganot, Annie; Phillips, Dawn; Woollacott, Ione O. C.; Bateman-House, Alison; Flanigan, Kevin M.; Goemans, Nathalie; Servais, Laurent; Guglieri, Michela; Mansfield, Carol
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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
err2025-08-23
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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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Treatment with UX111 gene therapy rapidly reduced heparansulfate (HS) exposure in cerebrospinalfluid (CSF) and improvedlong-term cognitive function in children withmucopolysaccharidosis type IIIA (MPS IIIA)
err2025-02-01
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PREAI
errLau, Heather A.; Patra, Kaushik; Wolf, Melissa; Smith, Nicholas J. C.; Couce, Maria L.; Rajan, Deepa; Truxal, Kristen; Castro Lopez, Maria Jose De; Fuller, Maria; Monteagudo-Vilavedra, Eines; Dunia Dougherty-De Miguel, Lucia; Riera, Mireia Del Toro; Flanigan, Kevin M.
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Gross motor delays in infants and young boys with Duchenne muscular dystrophy杜氏肌营养不良症婴儿和年幼男孩的大运动发育迟缓
err2024-12-08
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errLowes, Linda P.; Reash, Natalie F.; Iammarino, Megan A.; Connolly, Anne M.; Pietruszewski, Lindsay; Smith, Melissa A.; Peng, Jing; Steiner, Christopher L.; Tsao, Chang-Yong; Waldrop, Megan A.; Flanigan, Kevin M.; Chagat, Shannon; Meyer, Alayne P.; Mendell, Jerry R.; Alfano, Lindsay N.
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Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease
err2024-06-01
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PREAI
errHerstine, Jessica A.; Chang, Pi -Kai; Chornyy, Sergiy; Stevenson, Tamara J.; Sunshine, Alex C.; Nokhrina, Ksenia; Rediger, Jessica; Wentz, Julia; Vetter, Tatyana A.; Scholl, Erika; Holaway, Caleb; Pyne, Nettie K.; Bratasz, Anna; Yeoh, Stewart; Flanigan, Kevin M.; Bonkowsky, Joshua L.; Bradbury, Allison M.
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Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study
err2024-03-08
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errOAAI
errNicolau, Stefan; Malhotra, Jyoti; Kaler, Maryann; Coxen, Pamela Magistrado; Iammarino, Megan A.; Reash, Natalie F.; Frair, Emma C.; Wijeratne, Saranga; Kelly, Benjamin J.; White, Peter; Lowes, Linda P.; Waldrop, Megan A.; Flanigan, Kevin M.
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Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease
err2024-02-13
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errOAAI
errMcDonald, Craig; Camino, Eric; Escandon, Rafael; Finkel, Richard S.; Fischer, Ryan; Flanigan, Kevin; Furlong, Pat; Juhasz, Rose; Martin, Ann S.; Villa, Chet; Sweeney, H. Lee
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A genome-wide association analysis of loss of ambulation in dystrophinopathy patients suggests multiple candidate modifiers of disease severity
err2023-03-20
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errFlanigan, Kevin M.; Waldrop, Megan A.; Martin, Paul T.; Alles, Roxane; Dunn, Diane M.; Alfano, Lindsay N.; Simmons, Tabatha R.; Moore-Clingenpeel, Melissa; Burian, John; Seok, Sang-Cheol; Weiss, Robert B.; Vieland, Veronica J.
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Interim results of Transpher A, a multicenter, single-dose clinical trial of UX111 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
err2023-02-01
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PREAI
errFlanigan, Kevin M.; Smith, Nicholas; Luz Couce, Maria; Rajan, Deepa; Truxal, Kristen; McBride, Kim L.; de Castro Lopez, Maria Jose; Fuller, Maria; Taylor, John; Del Campo, Ana B.; Grachev, Igor; Lau, Heather
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Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy
err2022-12-01
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errGushchina, Liubov V.; Vetter, Tatyana A.; Frair, Emma C.; Bradley, Adrienne J.; Grounds, Kelly M.; Lay, Jacob W.; Huang, Nianyuan; Suhaiba, Aisha; Schnell, Frederick J.; Hanson, Gunnar; Simmons, Tabatha R.; Wein, Nicolas; Flanigan, Kevin M.
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Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy杜氏和贝克尔肌营养不良症的内含子突变和早期转录终止
err2022-03-07
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errOAAI
errWaldrop, Megan A.; Moore, Steven A.; Mathews, Katherine D.; Darbro, Benjamin W.; Medne, Livja; Finkel, Richard; Connolly, Anne M.; Crawford, Thomas O.; Drachman, Daniel; Wein, Nicolas; Habib, Ali A.; Krzesniak-Swinarska, Monika A.; Zaidman, Craig M.; Collins, James J.; Jokela, Manu; Udd, Bjarne; Day, John W.; Ortiz-Guerrero, Gloria; Statland, Jeff; Butterfield, Russell J.; Dunn, Diane M.; Weiss, Robert B.; Flanigan, Kevin M.
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Phenotypic Spectrum of Dystrophinopathy Due to Duchenne Muscular Dystrophy Exon 2 Duplications
err2022-02-15
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errZambon, Alberto A.; Waldrop, Megan A.; Alles, Roxane; Weiss, Robert B.; Conroy, Sara; Moore-Clingenpeel, Melissa; Previtali, Stefano; Flanigan, Kevin M.
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Interim results of Transpher A, a multicentre, single-dose, phase 1/2 clinical trial of ABO-102 investigational gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
err2022-02-01
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PREAI
errFlanigan, Kevin M.; Smith, Nicholas J. N.; Couce, Maria L.; Escolar, Maria; Truxal, Kristen V.; McBride, Kim L.; de Castro, Maria J.; Fuller, Maria; Paneda, A.; Ruiz, J.; del Campo, A. B.; Grachev, I.
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Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies
err2021-12-28
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errVetter, Tatyana A.; Nicolau, Stefan; Bradley, Adrienne J.; Frair, Emma C.; Flanigan, Kevin M.
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