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Validity and Reliability of Clinical and Patient-Reported Outcomes in Multisystem Proteinopathy 1 Alfano, Lindsay N.; Iammarino, Megan A.; Reash, Natalie F.; Lowes, Linda P.; Pietruszewski, Lindsay; Adderley, Kathleen; Humphrey, Lauren; Knight, Audrey B.; Steiner, Christopher L.; Smith, Melissa A.; Sahenk, Zarife; Connolly, Anne M.; Almomen, Momen; D'Ambrosio, Eleonora S.; Peck, Nathan; Peck, Allison 分享 收藏
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Lesion level-dependent systemic muscle wasting after spinal cord injury is mediated by glucocorticoid signaling in mice 糖皮质激素信号介导小鼠脊髓损伤后病变水平依赖性全身性肌肉萎缩 Harrigan, Markus E.; Filous, Angela R.; Vadala, Christopher P.; Webb, Amy; Pietrzak, Maciej; Sahenk, Zarife; Pruss, Harald; Reiser, Peter J.; Popovich, Phillip G.; Arnold, W. David; Schwab, Jan M. 分享 收藏
Clinical Classification of Variants in the Valosin-Containing Protein Gene Associated With Multisystem Proteinopathy Schiava, Marianela; Ikenaga, Chiseko; Topf, Ana; Caballero-Avila, Marta; Chou, Tsui-Fen; Li, Shan; Wang, Feng; Daw, Jil; Stojkovic, Tanya; Villar-Quiles, Rocio; Nishino, Ichizo; Inoue, Michio; Nishimori, Yukako; Saito, Yoshihiko; Katsuno, Masahisa; Noda, Seiya; Ito, Chihiro; Otsuka, Mieko; Nahir, Sruthi; Manousakis, Georgios; Walk, David; Quinn, Colin; Alfano, Lindsay; Sahenk, Zarife; Tasca, Giorgio; Monforte, Mauro; Sabatelli, Mario; Bisogni, Giulia; Oldfors, Anders; Rydeliu, Anna; Pal, Endre; Paradas, Carmen; Velez, Beatriz; De Bleecker, Jan L.; Farugia, Maria Elena; Longman, Cheryl; Harms, Matthew B.; Ralston, Stuart; Zanoteli, Edmar; da Silva, Andre Macedo Serafim; Sotoca, Javier; Juntas-Morales, Raul; Bevilacqua, Jorge; Balart, Mireya; Talbot, Stuart; Straub, Volker; Guglieri, Michela; Marini-Bettolo, Chiara; Diaz-Manera, Jordi; Weihl, Conrad Chris 分享 收藏
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Long-term Safety and Efficacy in Patients with DMD 4 Years Post-Treatment with Delandistrogene Moxeparvovec (SRP-9001) in a Phase 1/2a Study Mendell, Jerry; Sahenk, Zarife; Lehman, Kelly; Lowes, Linda; Reash, Natalie; Iammarino, Megan; Alfano, Lindsay; Lewis, Sarah; Church, Kathleen; Shell, Richard; Potter, Rachael; Griffin, Danielle; Hogan, Mark; Wang, Shufang; Mason, Stefanie; Darton, Eddie; Rodino-Klapac, Louise 分享 收藏
A Phase 2 Clinical Trial Evaluating the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Patients with Duchenne Muscular Dystrophy (DMD) Mendell, Jerry; Shieh, Perry; Sahenk, Zarife; Lehman, Kelly; Lowes, Linda; Reash, Natalie; Iammarino, Megan; Alfano, Lindsay; Sabo, Brenna; Woods, Jeremy; Skura, Christy; Mao, Howard; Staudt, Loretta; Potter, Rachael; Griffin, Danielle; Lewis, Sarah; Wang, Shufang; Singh, Tejdip; Rodino-Klapac, Louise 分享 收藏
A phase 2 clinical trial evaluating the safety and efficacy of delandistrogene moxeparvovec for treating patients with Duchenne muscular dystrophy 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 分享 收藏
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PUMILIO hyperactivity drives premature aging of Norad-deficient mice Kopp, Florian; Elguindy, Mahmoud M.; Yalvac, Mehmet E.; Zhang, He; Chen, Beibei; Gillett, Frank A.; Lee, Sungyul; Sivakumar, Sushama; Yu, Hongtao; Xie, Yang; Mishra, Prashant; Sahenk, Zarife; Mendell, Joshua T. 分享 收藏
Eteplirsen treatment for Duchenne muscular dystrophy Exon skipping and dystrophin production Charleston, Jay S.; Schnell, Frederick J.; Dworzak, Johannes; Donoghue, Cas; Lewis, Sarah; Chen, Lei; Young, G. David; Milici, Anthony J.; Voss, Jon; DeAlwis, Uditha; Wentworth, Bruce; Rodino-Klapac, Louise R.; Sahenk, Zarife; Frank, Diane; Mendell, Jerry R. 分享 收藏
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes Mendell, Jerry R.; Sahenk, Zarife; Al-Zaidy, Samiah; Rodino-Klapac, Louise R.; Lowes, Linda P.; Alfano, Lindsay N.; Berry, Katherine; Miller, Natalie; Yalvac, Mehmet; Dvorchik, Igor; Moore-Clingenpeel, Melissa; Flanigan, Kevin M.; Church, Kathleen; Shontz, Kim; Curry, Choumpree; Lewis, Sarah; McColly, Markus; Hogan, Mark J.; Kaspar, Brian K. 分享 收藏
Follistatin Gene Therapy Improves Six Minute Walk Distance in Sporadic Inclusion Body Myositis (sIBM) Mendell, Jerry R.; Sahenk, Zarife; Hogan, Mark; Al-Zaidy, Samiah; Flanigan, Kevin; Rodino-Klapac, Louise R.; McColly, Markus; Church, Kathleen; Lewis, Sarah; Lowes, Linda; Alfano, Lindsay; Berry, Katherine; Miller, Natalie; Dvorchik, Igor; Moore-Clingenpeel, Melissa; Kaspar, Brian K. 分享 收藏
Defective membrane fusion and repair in Anoctamin5-deficient muscular dystrophy Griffin, Danielle A.; Johnson, Ryan W.; Whitlock, Jarred M.; Pozsgai, Eric R.; Heller, Kristin N.; Grose, William E.; Arnold, W. David; Sahenk, Zarife; Hartzell, H. Criss; Rodino-Klapac, Louise R. 分享 收藏
Eteplirsen, a Phosphorodiamidate Morpholino Oligomer (PMO) for the Treatment of Duchenne Muscular Dystrophy (DMD): 168 Week Update on Six-Minute Walk Test (6MWT), Pulmonary Function Testing (PFT), and Safety Mendell, Jerry R.; Rodino-Klapac, Louise; Sahenk, Zarife; Rouch, Kandice; Bird, Lauren; Lowes, Linda; Alfano, Lindsay; Berry, Katherine; Lewis, Sarah; Shontz, Kim; Flanigan, Kevin; Shilling, Christopher; Duda, Petra; Saoud, Jay 分享 收藏
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