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

Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial

普利多吡啶治疗肌萎缩侧索硬化症:HEALEY ALS平台试验

Shefner, Jeremy M; Oskarsson, Björn; Macklin, Eric A; Chibnik, Lori B; Quintana, Melanie; Saville, Benjamin R; Detry, Michelle A; Vestrucci, Matteo; Marion, Joe; McGlothlin, Anna; Heiman-Patterson, Terry; Chase, Marianne; Pothier, Lindsay; Harkey, Brittney A; Yu, Hong; Sherman, Alexander V; Hall, Meghan; Kittle, Gale; Berry, James D; 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; Maragakis, Nicholas J; Simmons, Zachary; Miller, Timothy M; Olney, Nicholas; Weiss, Michael D; Goutman, Stephen A; Fernandes, Joseph Americo; Jawdat, Omar; Owegi, Margaret Ayo; Foster, Laura A; Vu, Tuan; Ilieva, Hristelina; Newman, Daniel S; Arcila-Londono, Ximena; Jackson, Carlayne E; Ladha, Shafeeq; Caress, James B; Swenson, Andrea; Peltier, Amanda; Lewis, Richard A; Fee, Dominic; Elliott, Matthew; Bedlack, Richard; Kasarskis, Edward J; Elman, Lauren; Rosenfeld, Jeffrey; Walk, David; McIlduff, Courtney; Twydell, Paul; Young, Eufrosina; Johnson, Kristin; Rezania, Kourosh; Goyal, Namita A; Cohen, Jeffrey A; Benatar, Michael; Jones, Vovanti; Shah, Jaimin; Beydoun, Said R; Wymer, James P; Zilliox, Lindsay; Nayar, Shakti; Pattee, Gary L; Martinez-Thompson, Jennifer; Leitner, Melanie L; Chen, Kelly; Goldberg, Y Paul; Cohen, Yael; Geva, Michal; Hayden, Michael R; Paganoni, Sabrina; Cudkowicz, Merit E

Verdiperstat in Amyotrophic Lateral Sclerosis: Results From the Randomized HEALEY ALS Platform Trial

Verdiperstat治疗肌萎缩侧索硬化症:来自随机HEALEY ALS平台试验的结果

Andrews, Jinsy; Paganoni, Sabrina; Macklin, Eric A; Chibnik, Lori B; Quintana, Melanie; Saville, Benjamin R; Detry, Michelle A; Vestrucci, Matteo; Marion, Joseph; McGlothlin, Anna; Young, Eufrosina; Chase, Marianne; Pothier, Lindsay; Harkey, Brittney; Yu, Hong; Sherman, Alex; Shefner, Jeremy; Hall, Meghan; Kittle, Gale; Connolly, Mariah R; Berry, James D; D'Agostino, Derek; Tustison, Eric; Giacomelli, Elisa; Scirocco, Erica; Alameda, Gustavo; Locatelli, Eduardo; Ho, Doreen; Quick, Adam; Heitzman, Daragh; Ajroud-Driss, Senda; Appel, Stanley H; Shroff, Sheetal; Katz, Jonathan; Felice, Kevin; Maragakis, Nicholas J; Simmons, Zachary; Goutman, Stephen A; Olney, Nicholas; Miller, Timothy; Fernandes, Joseph Americo; Ilieva, Hristelina; Jawdat, Omar; Weiss, Michael D; Foster, Laura; Vu, Tuan; Ladha, Shafeeq; Owegi, Margaret Ayo; Newman, Daniel S; Arcila-Londono, Ximena; Jackson, Carlayne E; Swenson, Andrea; Heiman-Patterson, Terry; Caress, James; Fee, Dominic; Peltier, Amanda; Lewis, Richard; Rosenfeld, Jeffrey; Walk, David; Johnson, Kristin; Elliott, Matthew; Kasarskis, Edward J; Rutkove, Seward; McIlduff, Courtney E; Bedlack, Richard; Elman, Lauren; Goyal, Namita A; Rezania, Kourosh; Twydell, Paul; Benatar, Michael; Glass, Jonathan; Cohen, Jeffrey A; Jones, Vovanti; Zilliox, Lindsay; Wymer, James P; Beydoun, Said R; Shah, Jaimin; Pattee, Gary L; Martinez-Thompson, Jennifer; Nayar, Shakti; Granit, Volkan; Donohue, Mary; Grossman, Katheryn; Campbell, Daniel J; Qureshi, Irfan A; Cudkowicz, Merit E; Babu, Suma

Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial

Zilucoplan治疗肌萎缩侧索硬化症的疗效和安全性:一项随机临床试验

Paganoni, Sabrina; Fournier, Christina N; Macklin, Eric A; Chibnik, Lori B; Quintana, Melanie; Saville, Benjamin R; Detry, Michelle A; Vestrucci, Matteo; Marion, Joe; McGlothlin, Anna; Ajroud-Driss, Senda; Chase, Marianne; Pothier, Lindsay; Harkey, Brittney A; Yu, Hong; Sherman, Alexander V; Shefner, Jeremy M; Hall, Meghan; Kittle, Gale; Berry, James D; Babu, Suma; Andrews, Jinsy; Dagostino, Derek; Tustison, Eric; Giacomelli, Elisa; Scirocco, Erica; Alameda, Gustavo; Locatelli, Eduardo; Ho, Doreen; Quick, Adam; Katz, Jonathan; Heitzman, Daragh; Appel, Stanley H; Shroff, Sheetal; Felice, Kevin; Maragakis, Nicholas J; Simmons, Zachary; Miller, Timothy M; Olney, Nicholas; Weiss, Michael D; Goutman, Stephen A; Fernandes, Joseph Americo; Jawdat, Omar; Owegi, Margaret Ayo; Foster, Laura A; Vu, Tuan; Ilieva, Hristelina; Newman, Daniel S; Arcila-Londono, Ximena; Jackson, Carlayne E; Ladha, Shafeeq; Heiman-Patterson, Terry; Caress, James B; Swenson, Andrea; Peltier, Amanda; Lewis, Richard; Fee, Dominic; Elliott, Matthew; Bedlack, Richard; Kasarskis, Edward J; Elman, Lauren; Rosenfeld, Jeffrey; Walk, David; McIlduff, Courtney; 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; Harvey, Brittany; Patel, Shital; Mahoney, Paul; Duda, Petra W; Cudkowicz, Merit E

Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial

COMET试验中,经过145周的治疗,评估了阿伐葡萄糖苷酶α对晚发型庞贝病患者的疗效和安全性。

Kishnani, Priya S; Díaz-Manera, Jordi; Illarioshkin, Sergey; van der Ploeg, Ans T; Clemens, Paula R; Day, John W; Toscano, Antonio; Kushlaf, Hani; Ladha, Shafeeq; Attarian, Shahram; Carvalho, Gerson; Kostera-Pruszczyk, Anna; Erdem-Özdamar, Sevim; Goker-Alpan, Ozlem; Mozaffar, Tahseen; Straub, Volker; Roberts, Mark; Haack, Kristina An; Huynh-Ba, Olivier; Tammireddy, Swathi; Periquet, Magali; Thibault, Nathan; Zhou, Tianyue; Dimachkie, Mazen M; Schoser, Benedikt

Multicenter Expanded Access Protocol for Research Through Access to Trehalose in People With Amyotrophic Lateral Sclerosis

通过获取海藻糖,为肌萎缩侧索硬化症患者开展研究的多中心扩大使用方案

Krivickas, Brooke; Scirocco, Erica; Giacomelli, Elisa; Sharma, Saloni; Benson, Molly; Keegan, Mackenzie; Kulesa-Kelley, Jennifer; Chibnik, Lori B; Casagrande, Gabriella; Heyd, Lindsay; Chase, Marianne; Drake, Kristin; Mohapatra, Silpa; Hagar, Jennifer Linn; Hasenoehrl, Meredith Gibbons; Dagostino, Derek; Sherman, Alexander V; Leite, Alex; Yu, Hong; Rosenthal, Jesse; Miller, Timothy; McCaffrey, Alexandra; Gwathmey, Kelly; Locatelli, Eduardo; Bayat, Elham; Heitzman, Daragh; Young, Eufrosina; Goyal, Namita A; Whitesell, Jackie; Felice, Kevin; Ilieva, Hristelina; Swenson, Andrea; Walk, David; Alameda, Gustavo; Foster, Laura; McIlduff, Courtney E; Walsh, Alison; Zilliox, Lindsay; Ajroud-Driss, Senda; Bodkin, Cynthia; Katz, Jonathan; Ladha, Shafeeq; Rivner, Michael; Rosow, Laura; Twydell, Paul; Wasiewski, Warren; Babu, Suma; Berry, James D; Paganoni, Sabrina

Serious Neurologic Adverse Events in Tofersen Clinical Trials for Amyotrophic Lateral Sclerosis

托弗森治疗肌萎缩侧索硬化症临床试验中发生的严重神经系统不良事件

Lovett, Alexandra; Chary, Sowmya; Babu, Suma; Bruneteau, Gaëlle; Glass, Jonathan D; Karlsborg, Merete; Ladha, Shafeeq; Mayl, Keith; McDermott, Christopher; Bucelli, Robert C; Chiò, Adriano; Ferguson, Toby A; Cochrane, Thos; Fradette, Stephanie; Smirnakis, Karen; Inra, Jennifer; Malek, Sohail; Fanning, Laura

Transthyretin abnormalities in amyotrophic lateral sclerosis: High molecular weight species in cerebrospinal fluid and stromal deposits in choroid plexus

肌萎缩侧索硬化症中的转甲状腺素蛋白异常:脑脊液中的高分子量物质和脉络丛基质沉积物

Collins, Mahlon; Kwapiszeski, Heather; An, Jiyan; Garcia, Deyri; Peller, Danielle; Ladha, Shafeeq; Bowser, Robert; Bakkar, Nadine

Safety and tolerability of tegoprubart in patients with amyotrophic lateral sclerosis: A Phase 2A clinical trial.

替戈普鲁巴特治疗肌萎缩侧索硬化症患者的安全性和耐受性:一项 2A 期临床试验

Perrin Steven, Ladha Shafeeq, Maragakis Nicholas, Rivner Michael H, Katz Jonathan, Genge Angela, Olney Nicholas, Lange Dale, Heitzman Daragh, Bodkin Cynthia, Jawdat Omar, Goyal Namita A, Bornstein Jeffrey D, Mak Carmen, Appel Stanley H, Paganoni Sabrina

Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial

阿伐葡萄糖苷酶α治疗晚发型庞贝病患者97周后的疗效和安全性:一项3期随机临床试验

Kishnani, Priya S; Diaz-Manera, Jordi; Toscano, Antonio; Clemens, Paula R; Ladha, Shafeeq; Berger, Kenneth I; Kushlaf, Hani; Straub, Volker; Carvalho, Gerson; Mozaffar, Tahseen; Roberts, Mark; Attarian, Shahram; Chien, Yin-Hsiu; Choi, Young-Chul; Day, John W; Erdem-Ozdamar, Sevim; Illarioshkin, Sergey; Goker-Alpan, Ozlem; Kostera-Pruszczyk, Anna; van der Ploeg, Ans T; An Haack, Kristina; Huynh-Ba, Olivier; Tammireddy, Swathi; Thibault, Nathan; Zhou, Tianyue; Dimachkie, Mazen M; Schoser, Benedikt