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

CNM-Au8 in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial

CNM-Au8 在肌萎缩侧索硬化症中的应用:HEALEY ALS 平台试验

Berry, James D; Maragakis, Nicholas J; Macklin, Eric A; Chibnik, Lori B; Quintana, Melanie; Saville, Benjamin R; Detry, Michelle A; Vestrucci, Matteo; Marion, Joseph; McGlothlin, Anna; Stommel, Elijah W; Chase, Marianne; Pothier, Lindsay; Harkey, Brittney A; Yu, Hong; Sherman, Alex; Shefner, Jeremy; Hall, Meghan; Kittle, Gale; Babu, Suma; Andrews, Jinsy; D'Agostino, Derek; Tustison, Eric; Scirocco, Erica; Giacomelli, Elisa; Alameda, Gustavo; Locatelli, Eduardo; Ho, Doreen; Quick, Adam; Ajroud-Driss, Senda; Katz, Jonathan; Heitzman, Daragh; Appel, Stanley H; Shroff, Sheetal; Felice, Kevin J; Simmons, Zachary; Miller, Timothy; Olney, Nicholas; Weiss, Michael D; Goutman, Stephen A; Fernandes, Joseph Americo Jr; Jawdat, Omar; Owegi, Margaret Ayo; Foster, Laura; Vu, Tuan; Ilieva, Hristelina; Newman, Daniel S; Arcila-Londono, Ximena; Jackson, Carlayne; Ladha, Shafeeq; Heiman-Patterson, Terry; Caress, James; Swenson, Andrea; Peltier, Amanda; Lewis, Richard; Fee, Dominic; Elliott, Matthew; Bedlack, Richard; Kasarskis, Edward J; Elman, Lauren; Rosenfeld, Jeffrey; Walk, David; McIlduff, Courtney E; Twydell, Paul; Young, Eufrosina; Johnson, Kristin; Rezania, Kourosh; Goyal, Namita A; Cohen, Jeffrey A; Benatar, Michael; Jones, Vovanti; Glass, Jonathan; Shah, Jaimin; Beydoun, Said R; Wymer, James P; Zilliox, Lindsay; Nayar, Shakti; Pattee, Gary L; Martinez-Thompson, Jennifer; Rynders, Austin; Evan, Jacob; Evan, Jeremy; Hartford, Alan; Sepassi, Marjan; Ho, Karen S; Glanzman, Robert; Greenberg, Benjamin; Hotchkin, Michael T; Paganoni, Sabrina; Cudkowicz, Merit E

Spinal Muscular Atrophy Functional Composite Score Revised (SMA-FCR) in Untreated and Nusinersen-Treated Patient Cohorts

脊髓性肌萎缩症功能综合评分修订版(SMA-FCR)在未治疗和接受Nusinersen治疗的患者队列中的比较

Pasternak, Amy; McDermott, Michael P; Montes, Jacqueline; Glanzman, Allan M; Coratti, Giorgia; Dunaway Young, Sally; Duong, Trinh Tina; Martens, William B; Day, John W; Zolkipli-Cunningham, Zarazuela; Sansone, Valeria Ada; D'Amico, Adele; Messina, Sonia; Bruno, Claudio; Mercuri, Eugenio; De Vivo, Darryl C; Darras, Basil T

Longitudinal Assessment of 4-Year HFMSE Changes in SMA II and III Patients Treated With Nusinersen

对接受诺西那生治疗的SMA II型和III型患者进行4年HFMSE评分变化的纵向评估

Coratti, Giorgia; Bovis, Francesca; Pane, Marika; Pasternak, Amy; Albamonte, Emilio; Mizzoni, Irene; Glanzman, Allan M; Morando, Simone; Montes, Jacqueline; Cavallina, Ilaria; Dunaway Young, Sally; Duong, Tina; Rolle, Enrica; Civitello, Matthew; De Sanctis, Roberto; Bravetti, Chiara; Ricci, Federica; Gadaleta, Giulio; Mongini, Tiziana; Sframeli, Maria; Pera, Maria Carmela; Messina, Sonia; D'Amico, Adele; Catteruccia, Michela; Brolatti, Noemi; Hirano, Michio; Zolkipli-Cunningham, Zarazuela; Darras, Basil T; Bertini, Enrico; Bruno, Claudio; Day, John; Sansone, Valeria A; Finkel, Richard S; Mercuri, Eugenio

Serial casting for contractures in SMA: consensus derived guidelines for treatment

脊髓性肌萎缩症挛缩的连续石膏固定:基于共识的治疗指南

Brown, Laurey; Hoffman, Katie; Corbo-Galli, Chiara; Kelley, Carolyn; Carry, Terri; Civitello, Matt; Coratti, Giorgia; DeSanctis, Roberto; Duong, Tina; Driscoll, Brigid; Flickinger, Jean; Glanzman, Allan M; Jones, Jennifer; Maczek, Elizabeth; Moat, Dionne; Montes, Jacqueline; Muni-Lofra, Robert; Nelson, Leslie; Pasternak, Amy; Valle, Melanie; Krosschell, Kristin J

Correction: Serial casting for contractures in SMA: consensus derived guidelines for treatment

更正:脊髓性肌萎缩症挛缩的连续石膏固定:治疗共识指南

Brown, Laurey; Hoffman, Katie; Corbo-Galli, Chiara; Kelley, Carolyn; Carry, Terri; Civitello, Matt; Coratti, Giorgia; DeSanctis, Roberto; Duong, Tina; Driscoll, Brigid; Flickinger, Jean; Glanzman, Allan M; Jones, Jennifer; Maczek, Elizabeth; Moat, Dionne; Montes, Jacqueline; Muni-Lofra, Robert; Nelson, Leslie; Pasternak, Amy; Valle, Melanie; Krosschell, Kristin J

Correction: Evidence of brain target engagement in Parkinson's disease and multiple sclerosis by the investigational nanomedicine, CNM- Au8, in the REPAIR phase 2 clinical trials

更正:在 REPAIR II 期临床试验中,研究性纳米药物 CNM-Au8 显示出与帕金森病和多发性硬化症脑靶点结合的证据

Ren, Jimin; Dewey, Richard B 3rd; Rynders, Austin; Evan, Jacob; Evan, Jeremy; Ligozio, Shelia; Ho, Karen S; Sguigna, Peter V; Glanzman, Robert; Hotchkin, Michael T; Dewey, Richard B Jr; Greenberg, Benjamin M

Nonverbal Cognitive Skills in Children With Aicardi Goutières Syndrome

艾卡迪-古蒂埃综合征患儿的非语言认知技能

Gavazzi, Francesco; Vaia, Ylenia; Woidill, Sarah; Formanowski, Brielle; Peixoto de Barcelos, Isabella; Sevagamoorthy, Anjana; Modesti, Nicholson B; Charlton, Lauren; Cusack, Stacy V; Vincent, Ariel; D'Aiello, Russell; Jawad, Abbas; Galli, Jessica; Varesio, Costanza; Fazzi, Elisa; Orcesi, Simona; Glanzman, Allan M; Lorch, Scott; DeMauro, Sara B; Guez-Barber, Danielle; Waldman, Amy T; Vanderver, Adeline; Adang, Laura A

Long-term natural history in type II and III spinal muscular atrophy: a 4-year international study on the Hammersmith Functional Motor Scale Expanded

II型和III型脊髓性肌萎缩症的长期自然病程:一项为期4年的Hammersmith功能运动量表扩展版国际研究

Coratti, Giorgia; Bovis, Francesca; Pera, Maria Carmela; Civitello, Matthew; Rohwer, Annemarie; Salmin, Francesca; Glanzman, Allan M; Montes, Jacqueline; Pasternak, Amy; De Sanctis, Roberto; Dunaway Young, Sally; Duong, Tina; Mizzoni, Irene; Milev, Evelin; Sframeli, Maria; Morando, Simone; Albamonte, Emilio; D'Amico, Adele; Catteruccia, Michela; Brolatti, Noemi; Pane, Marika; Scoto, Mariacristina; Messina, Sonia; Escudero, Jesica Exposito; De Waele, Liesbeth; Hirano, Michio; Zolkipli-Cunningham, Zarazuela; Darras, Basil T; Bertini, Enrico; Nascimiento Osorio, Andres; Bruno, Claudio; Goemans, Natalie; Sansone, Valeria A; Day, John; Baranello, Giovanni; Muntoni, Francesco; Finkel, Richard; Mercuri, Eugenio

Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3

在未经治疗的2型和3型脊髓性肌萎缩症患者队列中,修订版哈默史密斯量表所反映的疾病轨迹

Wolfe, Amy; Stimpson, Georgia; Ramsey, Danielle; Coratti, Giorgia; Dunaway Young, Sally; Mayhew, Anna; Pane, Marika; Rohwer, Annemarie; Muni Lofra, Robert; Duong, Tina; O'Reilly, Emer; Milev, Evelin; Civitello, Matthew; Sansone, Valeria A; D'Amico, Adele; Bertini, Enrico; Messina, Sonia; Bruno, Claudio; Albamonte, Emilio; Mazzone, Elena; Main, Marion; Montes, Jacqueline; Glanzman, Allan M; Zolkipli-Cunningham, Zarazuela; Pasternak, Amy; Marini-Bettolo, Chiara; Day, John W; Darras, Basil T; De Vivo, Darryl C; Baranello, Giovanni; Scoto, Mariacristina; Finkel, Richard S; Mercuri, Eugenio; Muntoni, Francesco

Beyond Contractures in Spinal Muscular Atrophy: Identifying Lower-Limb Joint Hypermobility

脊髓性肌萎缩症的挛缩之外:识别下肢关节过度活动

Harding, Elizabeth R; Kanner, Cara H; Pasternak, Amy; Glanzman, Allan M; Dunaway Young, Sally; Rao, Ashwini K; McDermott, Michael P; Zolkipli-Cunningham, Zarazuela; Day, John W; Finkel, Richard S; Darras, Basil T; De Vivo, Darryl C; Montes, Jacqueline