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Natural history of limb girdle muscular dystrophy R1 (LGMDR1): a GRASP consortium study 肢带型肌营养不良R1（LGMDR1）的自然史：一项GRASP联盟研究 Hunn, Stephanie M.; Findlay, Andrew R.; 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
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Framing childhood-onset facioscapulohumeral dystrophy: from first symptoms to future trials 儿童期起病的面肩肱型肌营养不良症：从首发症状到未来试验 Dijkstra, Jildou N.; Erasmus, Corrie E.; Laurian, Pierre; Roets, Ally; Dodd, Matthew; Irving, Kathryn; Moreno, Cristiane Araujo Martins; Woodcock, Ian R.; Voermans, Nicol C.; Willis, Tracey; Mathews, Katherine; de Valle, Katy 分享 收藏
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Propensity-matched analysis comparing omaveloxolone treatment to Friedreich ataxia natural history data: a plain language summary 倾向性匹配分析比较奥马波唑醇治疗与弗里德赖希共济失调自然史数据：一份通俗语言摘要 Lynch, David R.; Goldsberry, Angie; Rummey, Christian; Farmer, Jennifer; Boesch, Sylvia; Delatycki, Martin B.; Giunti, Paola; Hoyle, J. Chad; Mariotti, Caterina; Mathews, Katherine D.; Nachbauer, Wolfgang; Perlman, Susan; Subramony, Sub H.; Wilmot, George; Zesiewicz, Theresa; Weissfeld, Lisa; Meyer, Colin 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
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
The ZFHX3 GGC Repeat Expansion Underlying Spinocerebellar Ataxia Type 4 has a Common Ancestral Founder 脊髓小脑性共济失调4型的ZFHX3 GGC重复扩增具有共同的祖先创始人 Chen, Zhongbo; Alvarez Jerez, Pilar; Anderson, Claire; Paucar, Martin; Lee, Jasmaine; Nilsson, Daniel; Macpherson, Hannah; Scardamaglia, Annarita; Montgomery, Kylie; Hardy, John; Singleton, Andrew B.; Tucci, Arianna; Mathews, Katherine D.; Fu, Ying-Hui; Engvall, Martin; Laffita-Mesa, Jose; Nennesmo, Inger; Wedell, Anna; Ptacek, Louis J.; Blauwendraat, Cornelis; Gustavsson, Emil K.; Svenningsson, Per; Ryten, Mina; Houlden, Henry 分享 收藏
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Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy Zaidman, Craig M.; Goedeker, Natalie L.; Aqul, Amal A.; Butterfield, Russell J.; Connolly, Anne M.; Crystal, Ronald G.; Godwin, Kara E.; Hor, Kan N.; Mathews, Katherine D.; Proud, Crystal M.; Smyth, Elizabeth Kula; Veerapandiyan, Aravindhan; Watkins, Paul B.; Mendell, Jerry R. 分享 收藏
Adaptive Long-Read Sequencing Reveals GGC Repeat Expansion in ZFHX3 Associated with Spinocerebellar Ataxia Type 4 Chen, Zhongbo; Gustavsson, Emil K.; Macpherson, Hannah; Anderson, Claire; Clarkson, Chris; Rocca, Clarissa; Self, Eleanor; Jerez, Pilar Alvarez; Scardamaglia, Annarita; Pellerin, David; Montgomery, Kylie; Lee, Jasmaine; Gagliardi, Delia; Luo, Huihui; Hardy, John; Polke, James; Singleton, Andrew B.; Blauwendraat, Cornelis; Mathews, Katherine D.; Tucci, Arianna; Fu, Ying-Hui; Houlden, Henry; Ryten, Mina; Ptacek, Louis J. 分享 收藏
The Early Care (0-3 Years) In Duchenne Muscular Dystrophy Meeting Report Armstrong, Niki; Apkon, Susan; Berggren, Kiera N.; Braun, Catherine; Ciafaloni, Emma; Connolly, Anne; Kennedy, Annie; Kuntz, Nancy; Mathews, Katherine; McGuire, Michelle; Parad, Richard; Scavina, Mena; Scharf, Rebecca J.; Waldrop, Megan 分享 收藏
Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data Lynch, David R.; Goldsberry, Angie; Rummey, Christian; Farmer, Jennifer; Boesch, Sylvia; Delatycki, Martin B.; Giunti, Paola; Hoyle, J. Chad; Mariotti, Caterina; Mathews, Katherine D.; Nachbauer, Wolfgang; Perlman, Susan; Subramony, S. H.; Wilmot, George; Zesiewicz, Theresa; Weissfeld, Lisa; Meyer, Colin 分享 收藏
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