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Nexiguran Ziclumeran Gene Editing in Hereditary ATTR with Polyneuropathy Gillmore, J.D.; Gane, E.; Taubel, J.; Pilebro, B.; Echaniz-Laguna, A.; Kao, J.; Litchy, W.; Shahda, S.; Haagensen, A.; Walsh, L.; et al. Nexiguran ziclumeran基因编辑在遗传性ATTR伴多发性神经病变中的应用。N. Engl. J. Med. 2025, 393, 1375–1386. [Google Scholar] [CrossRef] Gillmore, Julian D.; Gane, Ed; Taubel, Jorg; Pilebro, Bjorn; Echaniz-Laguna, Andoni; Kao, Justin; Litchy, William; Shahda, Safi; Haagensen, Alexandra; Walsh, Liron; Smith, Derek; Kachadourian, Jessica; Ward, Jonathan H.; Lebwohl, David; Zhu, Peijuan; Xu, Yuanxin; Leung, Adia; Sonderfan, Alison; Gutstein, David E.; Manvelian, Garen; Adams, David 分享 收藏
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Unraveling calcium dysregulation and autoimmunity in immune mediated rippling muscle disease (vol 13, 11, 2025) 解析免疫介导的肌肉颤搐病中的钙离子失调与自身免疫 (vol 13, 11, 2025) Nath, SR; Dasgupta, A; Dubey, D; Kokesh, E; Beecher, G; Fadra, N; Liewluck, T; Pittock, S; Doles, JD; Litchy, W; Milone, M 分享 收藏
High-Throughput Immunoassays for Cavin-4 IgG: A Diagnostic Tool for Immune-Mediated Rippling Muscle Disease LaFrance-Corey, Reghann G.; Kherbek, Haidara; Harinesan, Nimalan; Milone, Margherita; Paramasivan, Naveen K.; Sarker, Pallab; Knight, Andrew M.; Karsten, Carley; Dasari, Surendra; Liewluck, Teerin; Litchy, William J.; Pittock, Sean J.; Mills, John R.; Dubey, Divyanshu 分享 收藏
Unraveling calcium dysregulation and autoimmunity in immune mediated rippling muscle disease Nath, Samir R.; Dasgupta, Aneesha; Dubey, Divyanshu; Kokesh, Eileen; Beecher, Grayson; Fadra, Numrah; Liewuck, Teerin; Pittock, Sean; Doles, Jason D.; Litchy, William; Milone, Margherita 分享 收藏
Compound Muscle Action Potential and Myosin-Loss Pathology in Patients With Critical Illness Myopathy Correlation and Prognostication Shelly, Shahar; Soontrapa, Pannathat; Madigan, Nicolas N.; Polzin, Michael J.; Singh, Tarun D.; Sista, Sri Raghav S.; Paul, Pritikanta; Braksick, Sherri A.; Liao, Bing; Windebank, Anthony J.; Boon, Andrea J.; Litchy, William J.; Milone, Margherita; Liewluck, Teerin 分享 收藏
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Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy Coelho, Teresa; Ando, Yukio; Benson, Merrill D.; Berk, John L.; Waddington-Cruz, Marcia; Dyck, Peter J.; Gillmore, Julian D.; Khella, Sami L.; Litchy, William J.; Obici, Laura; Monteiro, Cecilia; Tai, Li-Jung; Viney, Nicholas J.; Buchele, Gustavo; Brambatti, Michela; Jung, Shiangtung W.; O'Dea, Louis St L.; Tsimikas, Sotirios; Schneider, Eugene; Geary, Richard S.; Monia, Brett P.; Gertz, Morie 分享 收藏
AANEM - IFCN glossary of terms in neuromuscular electrodiagnostic medicine and ultrasound Dengler, Reinhard; de Carvalho, Mamede; Shahrizaila, Nortina; Nodera, Hiroyuki; Vucic, Steve; Grimm, Alexander; Padua, Luca; Schreiber, Stefanie; Kneiser, Mary K.; Hobson-Webb, Lisa D.; Boon, Andrea J.; Smith, Benn E.; Litchy, William J.; Li, Yuebing; Lenihan, Michael; Thompson, V. Brandon; Stalberg, Erik; Sanders, Donald B.; Kincaid, John C. 分享 收藏
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Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis Benson, M. D.; Waddington-Cruz, M.; Berk, J. L.; Polydefkis, M.; Dyck, P. J.; Wang, A. K.; Plante-Bordeneuve, V.; Barroso, F. A.; Merlini, G.; Obici, L.; Scheinberg, M.; Brannagan, T. H., III; Litchy, W. J.; Whelan, C.; Drachman, B. M.; Adams, D.; Heitner, S. B.; Conceicao, I.; Schmidt, H. H.; Vita, G.; Campistol, J. M.; Gamez, J.; Gorevic, P. D.; Gane, E.; Shah, A. M.; Solomon, S. D.; Monia, B. P.; Hughes, S. G.; Kwoh, T. J.; McEvoy, B. W.; Jung, S. W.; Baker, B. F.; Ackermann, E. J.; Gertz, M. A.; Coelho, T. 分享 收藏
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