未登录 Establishing biomarkers and clinical endpoints in myotonic dystrophy type 1 (END-DM1): Protocol of an international natural history study 在强直性肌营养不良1型中建立生物标志物和临床终点(END-DM1)：一项国际性自然病史研究的方案 Mul, Karlien; Eichinger, Kate; Hung, Man; Sansone, Valeria A.; Gagnon, Cynthia; Subramony, Sub; Roxburgh, Richard H.; Hamel, Johanna; Statland, Jeffrey M.; Elsheikh, Bakri; Turner, Chris; Sampson, Jacinda; Ragole, Thomas; Matthews, Emma; Schoser, Benedikt; Swenson, Andrea; Laverty, Chamindra; Shieh, Perry; Greene, Ericka P.; Takahashi, Masanori; Wicklund, Matthew; Dekdebrun, Jeanne; Raymond, Jennifer; Despain, Erin; Thornton, Charles A.; Johnson, Nicholas E. 分享 收藏
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Clinical Trial Readiness in Limb Girdle Muscular Dystrophy R1 (LGMDR1): A GRASP Consortium Study Hunn, Stephanie M.; Alfano, Lindsay N.; Jones, Aileen; Butler, Amanda; Lowes, Linda P.; Iammarino, Megan A.; Reash, Natalie F.; Pietruszewski, Lindsay; Sasidharan, Sandhya; Currence, Melissa; Statland, Jeffrey M.; Strahler, Talia; Will, Robert; Wicklund, Matthew; Dixon, Stacy; Augsburger, Renee; Mozaffar, Tahseen; Laubscher, Katie M.; Mockler, Shelley R. H.; Mathews, Katherine D.; Stinson, Nikia; Leung, Doris G.; Stark, Molly M.; Horton, Rebecca A.; Kang, Peter B.; James, Meredith K.; Clause, Amanda; Weihl, Conrad C.; Johnson, Nicholas E. 分享 收藏
Strength and functional correlates of reachable workspace in facioscapulohumeral muscular dystrophy 面肩肱型肌营养不良症中可达工作空间的力量和功能相关性 Wang, Leo H.; Hatch, Maya N.; Mcdermott, Michael P.; Martens, William B.; Eichinger, Katy; Lewis, Leann; Walker, Michaela; Leung, Doris G.; Wagner, Kathryn R.; Sacconi, Sabrina; Mul, Karlien; Shieh, Perry B.; Elsheikh, Bakri; Butterfield, Russell J.; Johnson, Nicholas E.; Sansone, Valeria; Han, Jay J.; Tawil, Rabi; Statland, Jeffrey M.; ReSolve Investigators FSHD CTRN 分享 收藏
Prospective observational study of FKRP-related limb-girdle muscular dystrophy R9: A GRASP consortium study Alfano, Lindsay N.; James, Meredith K.; Petersen, Kristine v; Rudolf, Karen; Vissing, John; Augsburger, Renee; Mozaffar, Tahseen; Jones, Aileen; Butler, Amanda; Laubscher, Katie M.; Mockler, Shelley R. H.; Mathews, Katherine D.; Iammarino, Megan A.; Reash, Natalie F.; Pietruszewski, Lindsay; Lowes, Linda P.; Strahler, Talia; Wicklund, Matthew; Hunn, Stephanie; Weihl, Conrad C.; Sasidharan, Sandhya; Currence, Melissa; Statland, Jeffrey M.; Stinson, Nikia; Holzer, Megan; Leung, Doris G.; Lott, Donovan J.; Kang, Peter B.; Holsten, Scott; Desai, Urvi; Johnson, Nicholas E.; GRASP LGMD Consortium 分享 收藏
Prognostic clinical and biological markers for amyotrophic lateral sclerosis disease progression: validation and implications for clinical trial design and analysis Benatar, Michael; Macklin, Eric A.; Malaspina, Andrea; Rogers, Mary-Louise; Hornstein, Eran; Lombardi, Vittoria; Renfrey, Danielle; Shepheard, Stephanie; Magen, Iddo; Cohen, Yahel; Granit, Volkan; Statland, Jeffrey M.; Heckmann, Jeannine M.; Rademakers, Rosa; McHutchison, Caroline A.; Petrucelli, Leonard; McMillan, Corey T.; Wuu, Joanne 分享 收藏
Resistance exercise in early-stage ALS patients, ALSFRS-R, Sickness Impact Profile ALS-19, and muscle transcriptome: a pilot study Jawdat, Omar; Rucker, Jason; Nakano, Tomoki; Takeno, Kotaro; Statland, Jeffery; Pasnoor, Mamatha; Dimachkie, Mazen M.; Sabus, Carla; Badawi, Yomna; Hunt, Suzanne L.; Tomioka, Naoko H.; Gunewardena, Sumedha; Bloomer, Clark; Wilkins, Heather M.; Herbelin, Laura; Barohn, Richard J.; Nishimune, Hiroshi 分享 收藏
A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot-Marie-Tooth type 1A (vol 16, 433, 2021) PXT3003治疗1A型charcot-marie-tooth的双盲，安慰剂对照，随机试验 (第16卷，433，2021) Attarian, Shahram; Young, Peter; Brannagan, Thomas H.; Adams, David; Van Damme, Philip; Thomas, Florian P.; Casanovas, Carlos; Kafaie, Jafar; Tard, Celine; Walter, Maggie C.; Pereon, Yann; Walk, David; Stino, Amro; de Visser, Marianne; Verhamme, Camiel; Amato, Anthony; Carter, Gregory; Magy, Laurent; Statland, Jeffrey M.; Felice, Kevin 分享 收藏
Regional and bilateral MRI and gene signatures in facioscapulohumeral dystrophy: implications for clinical trial design and mechanisms of disease progression Wong, Chao-Jen; Friedman, Seth D.; Snider, Lauren; Bennett, Sean R.; Jones, Takako, I; Jones, Peter L.; Shaw, Dennis W. W.; Blemker, Silvia S.; Riem, Lara; Ducharme, Olivia; Lemmers, Richard J. F. L.; van der Maarel, Silvere M.; Wang, Leo H.; Tawil, Rabi; Statland, Jeffrey M.; Tapscott, Stephen J. 分享 收藏
Temporal course of cognitive and behavioural changes in motor neuron diseases McHutchison, Caroline A.; Wuu, Joanne; McMillan, Corey T.; Rademakers, Rosa; Statland, Jeffrey; Wu, Gang; Rampersaud, Evadnie; Myers, Jason; Hernandez, Jessica P.; Abrahams, Sharon; Benatar, Michael 分享 收藏
Autosomal dominant in cis D4Z4 repeat array duplication alleles in facioscapulohumeral dystrophy Lemmers, Richard J. L. F.; Butterfield, Russell; van der Vliet, Patrick J.; de Bleecker, Jan L.; van der Pol, Ludo; Dunn, Diane M.; Erasmus, Corrie E.; D'Hooghe, Marc; Verhoeven, Kristof; Balog, Judit; Bigot, Anne; van Engelen, Baziel; Statland, Jeffrey; Bugiardini, Enrico; van der Stoep, Nienke; Evangelista, Teresinha; Marini-Bettolo, Chiara; van den Bergh, Peter; Tawil, Rabi; Voermans, Nicol C.; Vissing, John; Weiss, Robert B.; van der Maarel, Silvere M. 分享 收藏
Twelve-month functional change in Limb Girdle Muscular Dystrophy R9 / 2I Johnson, Nicholas; Bates, Kameron; Xie, Han; Alfano, Lindsay; Vissing, John; Mozaffar, Tahseen; Mathews, Katherine; Lowes, Linda; Wicklund, Matthew; Weihl, Conrad; Leung, Doris; Kang, Peter; Zingariello, Carla; Desai, Urvi; DeSpain, Erin; Romain, Mary St.; Statland, Jeffrey 分享 收藏
Safety and Efficacy of Ataluren in nmDMD Patients from Study 041, a Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial McDonald, Craig M.; Wu, Shiwen; Gulati, Sheffali; Komaki, Hirofumi; Escobar, Rosa E.; Kostera-Pruszczyk, Anna; Vlodavets, Dmitry; Chae, Jong-Hee; Jong, Yuh-Jyh; Karachunski, Peter; Statland, Jeffrey; Lorentzos, Michelle; Penematsa, Vinay; Chou, Connie; Lin, Min; Werner, Christian; Trifillis, Panayiota; Gordon, Gregory; Koladicz, Karyn; Mastrandrea, Nicholas; Klein, Matthew 分享 收藏
Preliminary Assessment of the Phase 1/2 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Patients with Myotonic Dystrophy Type 1 (DM1) (MARINA) 1/2期临床试验的初步评估，评估静脉注射AOC 1001对1型强直性肌营养不良 (DM1) 成年患者的安全性，耐受性，药代动力学和药效学 (MARINA) Johnson, Nicholas; Day, John; Hamel, Johanna; Thornton, Charles; Subramony, S.; Soltanzadeh, Payam; Statland, Jeffrey; Arnold, William; Wicklund, Matthew; Ditrapani, Kelly; Heusner, Carrie; Chen, Chao-Yin; McEvoy, Bradley; Zhu, Yiming; Tai, Li-Jung; Ackermann, Elizabeth 分享 收藏
The FSHD Composite Outcome Measure (FSHD-COM) is Reliable, Valid, and Measures Disease Progression Eichinger, Katy; McDermott, Michael; Higgs, Kiley; Walker, Michaela; Lewis, Leann; Martens, William; Leung, Doris; Stinson, Nikia; Holzer, Megan M.; Sacconi, Sabrina; Garcia, Jeremy; Benito, Victor De Paz; Mul, Karlien; Sansone, Valeria; Carraro, Elena; Becchiati, Stefano; Frisoni, Maria Chiara; Wang, Leo; Kieu, Catherine; Shieh, Perry; Skura, Christy; Elsheikh, Bakri; Kelly, Kristina; Jaworek, Andrea; LoRusso, Samantha; Butterfield, Russell; Wilson, Amelia; McIntyre, Melissa; Johnson, Nicholas; Butler, Amanda; Jones, Aileen; Hayes, Melissa; Baker, Lindsay; Sasidharan, Sandhya; Tawil, Alrabi; Statland, Jeffrey 分享 收藏
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Motor Outcomes to Validate Evaluations in Facioscapulohumeral muscular dystrophy (MOVE FSHD): Preliminary Baseline Characteristics 验证面肩肱型肌营养不良症 (MOVE FSHD) 评估的运动结果: 初步基线特征 Walker, Michaela; Butterfield, Russell; Day, John; Eichinger, Katy; Elsheikh, Bakri; Friedman, Seth; Genge, Angela; Higgs, Kiley; Johnson, Nicholas; Jones, Peter; Leung, Doris; Lewis, Leann; Lochmuller, Hanns; O'Ferrall, Erin; Martens, William; Shaw, Dennis; Shieh, Perry; Subramony, S.; Trivedi, Jaya; Wang, Leo; Wicklund, Matthew; Tawil, Alrabi; Statland, Jeffrey 分享 收藏
MANOEUVRE Study Design: A Study of GYM329 (RO7204239) in Patients with Facioscapulohumeral Muscular Dystrophy Statland, Jeffrey; Eichinger, Katy; Morrow, Jasper; Tasca, Giorgio; Vissing, John; Dodman, Angela; Jaber, Birgit; Kletzl, Heidemarie; McIver, Tammy; Scalco, Renata; Yeung, Wai Yin; Gaki, Eleni; Wagner, Kathryn 分享 收藏
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