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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 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. 分享 收藏
Combination disease-modifying treatment in spinal muscular atrophy: A proposed classification Proud, Crystal M.; Mercuri, Eugenio; Finkel, Richard S.; Kirschner, Janbernd; De Vivo, Darryl C.; Muntoni, Francesco; Saito, Kayoko; Tizzano, Eduardo F.; Desguerre, Isabelle; Quijano-Roy, Susana; Benguerba, Kamal; Raju, Dheeraj; Faulkner, Eric; Servais, Laurent 分享 收藏
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Treatments and Outcomes for Patients with Spinal Muscular Atrophy Type 2: Findings from RESTORE Registry Servais, Laurent; Benguerba, Kamal; De Vivo, Darryl C.; Kirschner, Jan; Muntoni, Francesco; Proud, Crystal; Tizzano, Eduardo; Quijano-Roy, Susana; Desguerre, Isabelle; Saito, Kyoko; Raju, Dheeraj; LaMarca, Nicole; Sun, Rui; Anderson, Fred; Faulkner, Eric; Finkel, Richard 分享 收藏
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Plasma ADAMTS13 activity and von Willebrand factor antigen and activity in patients with subarachnoid haemorrhage Kumar, Monisha; Cao, Wenjing; Pham, Huy P.; Raju, Dheeraj; Nawalinski, Kelsey; Frangos, Suzanne; Kung, David; Zager, Eric E.; Kasner, Scott E.; Levine, Joshua M.; Zheng, X. Long 分享 收藏
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