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

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

Delandistrogene Moxeparvovec 治疗可活动杜氏肌营养不良症患者的两年疗效:3 期 EMBARK 试验

Mendell, Jerry R; Muntoni, Francesco; McDonald, Craig M; Mercuri, Eugenio M; Ciafaloni, Emma; Komaki, Hirofumi; Leon-Astudillo, Carmen; Nascimento, Andrés; Proud, Crystal; Schara-Schmidt, Ulrike; Veerapandiyan, Aravindhan; Zaidman, Craig M; Furgerson, Matthew; Ding, Kai; Singh, Preeti; Potter, Rachael; Asher, Damon R; Murphy, Alexander P; Reid, Carol; Hooper, Gregory; Torre, Carmen O; Manfrini, Marianna; Rodino-Klapac, Louise R

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.

AAV基因疗法治疗杜氏肌营养不良症:EMBARK 3期随机试验

Mendell Jerry R, Muntoni Francesco, McDonald Craig M, Mercuri Eugenio M, Ciafaloni Emma, Komaki Hirofumi, Leon-Astudillo Carmen, Nascimento Andrés, 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

Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study

静脉注射奥那西姆诺基因阿贝帕沃韦治疗脊髓性肌萎缩症患儿的安全性和有效性:3b期SMART研究

McMillan, Hugh J; Baranello, Giovanni; Farrar, Michelle A; Zaidman, Craig M; Moreno, Teresa; De Waele, Liesbeth; Jong, Yuh-Jyh; Laugel, Vincent; Quijano-Roy, Susana; Mercuri, Eugenio; Chien, Yin-Hsiu; Straub, Volker; Darras, Basil T; Seibert, Julia; Bernardo Escudero, Roberto; Alecu, Iulian; Freischläger, Frank; Muntoni, Francesco

Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy

对接受 delandistrogene moxeparvovec 基因治疗后发生的转基因导向免疫介导性肌炎进行免疫学研究

Potter, Rachael A; Moeller, Ida H; Khan, Sohrab; Haegel, Hélène; 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

Effects of in vivo Lewy body pathology on neuropsychiatric symptoms across the Alzheimer's disease continuum

体内路易体病理对阿尔茨海默病全程神经精神症状的影响

Zaidman, Craig M; Connolly, Anne M; Malkus, Elizabeth C; Florence, Julaine M; Pestronk, Alan; Leffa, Douglas Teixeira; Povala, Guilherme; Ferreira, Pamela CL; Ferrari‐Souza, João Pedro; Bauer‐Negrini, Guilherme; Rodrigues, Matheus Scarpatto; Amaral, Livia; Lussier, Firoza Z; Medeiros, Marina Scop; Soares, Carolina; Macedo, Arthur C; Therriault, Joseph; Rosa‐Neto, Pedro; Tudorascu, Dana L; Zimmer, Eduardo R; Bellaver, Bruna; Pascoal, Tharick A

Immune-mediated myositis following gene therapy for Duchenne muscular dystrophy: a case report

杜氏肌营养不良症基因治疗后免疫介导性肌炎:病例报告

Iannaccone, Susan T; Cai, Chunyu; Rhem, Brittney; Batley, Kaitlin; Rajaram, Veena; Greenberg, Benjamin M; Dharia, Sachi; Zaidman, Craig M

Newborn Screening for Spinal Muscular Atrophy: Variations in Practice and Early Management of Infants with Spinal Muscular Atrophy in the United States

美国新生儿脊髓性肌萎缩症筛查:实践差异及脊髓性肌萎缩症患儿早期管理

Zaidman, Craig M; Crockett, Cameron D; Wedge, Ethan; Tabatabai, Grace; Goedeker, Natalie

Outcomes of early-treated infants with spinal muscular atrophy: A multicenter, retrospective cohort study

早期治疗脊髓性肌萎缩症患儿的预后:一项多中心回顾性队列研究

Goedeker, Natalie L; Rogers, Amanda; Fisher, Mark; Arya, Kapil; Brandsema, John F; Farah, Hiba; Farrar, Michelle A; Felker, Marcia V; Gibbons, Melissa; Hamid, Omer Abdul; Harmelink, Matthew; Herbert, Karen; Kichula, Elizabeth; King, Kiana; Lakhotia, Arpita; Lee, Bo Hoon; Kuntz, Nancy L; Parsons, Julie; Rehborg, Rebecca; Veerapaniyan, Aravindhan; Zaidman, Craig M

Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy

通过测量杜氏肌营养不良症患者体内总结合抗体来评估rAAVrh74基因治疗载体的血清阳性率

Goedeker, Natalie L; Dharia, Sachi D; Griffin, Danielle A; Coy, Jesantha; Truesdale, Todd; Parikh, Rajan; Whitehouse, Kasen; Santra, Sourav; Asher, Damon R; Zaidman, Craig M

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study

Viltolarsen治疗杜氏肌营养不良症男孩的疗效和安全性:来自2期开放标签4年扩展研究的结果

Clemens, Paula R; Rao, Vamshi K; Connolly, Anne M; Harper, Amy D; Mah, Jean K; McDonald, Craig M; Smith, Edward C; Zaidman, Craig M; Nakagawa, Tomoyuki; Hoffman, Eric P