未登录 Model-Informed Precision Dosing Guidance of Ethosuximide Developed from a Randomized Controlled Clinical Trial of Childhood Absence Epilepsy Mizuno, Kana V.; Capparelli, Edmund; Fukuda, Tsuyoshi; Dong, Min C.; Adamson, Peter L.; Blumer, Jeffery; Cnaan, Avital O.; Clark, Peggy D.; Reed, Michael; Shinnar, Shlomo A.; Vinks, Alexander A.; Glauser, Tracy; Childhood Absence Epilepsy Study Grp, Childhood Absence Epilepsy Study Grp 分享 收藏
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function Hoffman, Eric P.; Schwartz, Benjamin D.; Mengle-Gaw, Laurel J.; Smith, Edward C.; Castro, Diana; Mah, Jean K.; McDonald, Craig M.; Kuntz, Nancy L.; Finkel, Richard S.; Guglieri, Michela; Bushby, Katharine; Tulinius, Mar; Nevo, Yoram; Ryan, Monique M.; Webster, Richard; Smith, Andrea L.; Morgenroth, Lauren P.; Arrieta, Adrienne; Shimony, Maya; Siener, Catherine; Jaros, Mark; Shale, Phil; McCall, John M.; Nagaraju, Kanneboyina; van den Anker, John; Conklin, Laurie S.; Cnaan, Avital; Gordish-Dressman, Heather; Damsker, Jesse M.; Clemens, Paula R. 分享 收藏
Assessment of disease progression in dysferlinopathy: A 1-year cohort study Moore, Ursula; Jacobs, Marni; James, Meredith K.; Mayhew, Anna G.; Fernandez-Torron, Roberto; Feng, Jia; Cnaan, Avital; Eagle, Michelle; Bettinson, Karen; Rufibach, Laura E.; Lofra, Robert Muni; Blamire, Andrew M.; Carlier, Pierre G.; Mittal, Plavi; Lowes, Linda Pax; Alfano, Lindsay; Rose, Kristy; Duong, Tina; Berry, Katherine M.; Montiel-Morillo, Elena; Pedrosa-Hernandez, Irene; Holsten, Scott; Sanjak, Mohammed; Ashida, Ai; Sakamoto, Chikako; Tateishi, Takayuki; Yajima, Hiroyuki; Canal, Aurelie; Ollivier, Gwenn; Decostre, Valerie; Mendez, Juan Bosco; Praxedes, Nieves Sanchez-Aguilera; Thiele, Simone; Siener, Catherine; Shierbecker, Jeanine; Florence, Julaine M.; Vandevelde, Bruno; DeWolf, Brittney; Hutchence, Meghan; Gee, Richard; Pruegel, Juliana; Maron, Elke; Hilsden, Heather; Lochmueller, Hanns; Grieben, Ulrike; Spuler, Simone; Rocha, Carolina Tesi; Day, John W.; Jones, Kristi J.; Bharucha-Goebel, Diana X.; Salort-Campana, Emmanuelle; Harms, Matthew; Pestronk, Alan; Krause, Sabine; Schreiber-Katz, Olivia; Walter, Maggie C.; Paradas, Carmen; Hogrel, Jean-Yves; Stojkovic, Tanya; Takeda, Shin'ichi; Mori-Yoshimura, Madoka; Bravver, Elena; Sparks, Susan; Diaz-Manera, Jordi; Bello, Luca; Semplicini, Claudio; Pegoraro, Elena; Mendell, Jerry R.; Bushby, Kate; Straub, Volker; Arrieta, Adrienne; Hwang, Esther; Lee, Elaine; Illa, Isabel; Gallardo, Eduard; Belmonte Jimeno, Izaskun; Llauger Rossello, Jaume; Harwick, Bruce; Sykes, Jackie; Yetter, Brent; Smith, Mark; Lapeyssonie, Bernard; Bendahan, David; Le Fur, Yann; Shahram, Attarian; Albane, Testot-Ferry; Coppenrath, Eva M.; Harris, Elizabeth; Guglieri, Michela; Evangelista, Teresinha; Murphy, Alex; Moat, Dionne; Hodgson, Tim; Wallace, Dorothy; Ward, Louise; Galley, Debra; Calore, Chiara; Stramare, Roberto; Rampado, Alessandro; Gidaro, Teresa; Turk, Suna; Servais, Laurent; Theis, Cyrille; Diabate, Oumar; Schimmoeller, Linda; Foster, Glenn; Carbonell, Pilar; Cabrera, Macarena; Morgado, Yolanda; Gala, Susana Rico; Perez, Jennifer; Sawyer, Anne Marie; Clarke, Nigel F.; Sandaradura, Sarah; Ghaoui, Roula; Cornett, Kayla; Miller, Claire; Foster, Sheryl; Peduto, Anthony; Sato, Noriko; Tamaru, Takeshi; Kobayashi, Yoko; Ashida, Ai; Nakayama, Takahiro; Segawa, Kazuhiko; Ohtaguro, Sachiko; Nakamura, Harumasa; Ohhata, Maki; Kimura, En; Endo, Makiko; Brody, Nora; Leach, Meganne E.; Toles, Allyn; Fricke, Stanley T.; Otero, Hansel J. 分享 收藏
A multinational study on motor function in early-onset FSHD Mah, Jean K.; Feng, Jia; Jacobs, Marni B.; Duong, Tina; Carroll, Kate; de Valle, Katy; Carty, Cara L.; Morgenroth, Lauren P.; Guglieri, Michela; Ryan, Monique M.; Clemens, Paula R.; Thangarajh, Mathula; Webster, Richard; Smith, Edward; Connolly, Anne M.; McDonald, Craig M.; Karachunski, Peter; Tulinius, Mar; Harper, Amy; Cnaan, Avital; Chen, Yi-Wen 分享 收藏
Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study McDonald, Craig M.; Henricson, Erik K.; Abresch, Richard T.; Duong, Tina; Joyce, Nanette C.; Hu, Fengming; Clemens, Paula R.; Hoffman, Eric P.; Cnaan, Avital; Gordish-Dressman, Heather 分享 收藏
Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study Moore, Ursula R.; Jacobs, Marni; Fernandez-Torron, Roberto; Jang, Jiji; James, Meredith K.; Mayhew, Anna; Rufibach, Laura; Mittal, Plavi; Eagle, Michelle; Cnaan, Avital; Carlier, Pierre G.; Blamire, Andrew; Hilsden, Heather; Lochmueller, Hanns; Grieben, Ulrike; Spuler, Simone; Rocha, Carolina Tesi; Day, John W.; Jones, Kristi J.; Bharucha-Goebel, Diana X.; Salort-Campana, Emmanuelle; Harms, Matthew; Pestronk, Alan; Krause, Sabine; Schreiber-Katz, Olivia; Walter, Maggie C.; Paradas, Carmen; Hogrel, Jean-Yves; Stojkovic, Tanya; Takeda, Shin'ichi; Mori-Yoshimura, Madoka; Bravver, Elena; Sparks, Susan; Diaz-Manera, Jordi; Bello, Luca; Semplicini, Claudio; Pegoraro, Elena; Mendell, Jerry R.; Bushby, Kate; Straub, Volker 分享 收藏
Pretreatment behavior and subsequent medication effects in childhood absence epilepsy Shinnar, Ruth C.; Shinnar, Shlomo; Cnaan, Avital; Clark, Peggy; Dlugos, Dennis; Hirtz, Deborah G.; Hu, Fengming; Liu, Chunyan; Masur, David; Weiss, Erica F.; Glauser, Tracy A. 分享 收藏
Pretreatment seizure semiology in childhood absence epilepsy Kessler, Sudha Kilaru; Shinnar, Shlomo; Cnaan, Avital; Dlugos, Dennis; Conry, Joan; Hirtz, Deborah G.; Hu, Fengming; Liu, Chunyan; Mizrahi, Eli M.; Moshe, Solomon L.; Clark, Peggy; Glauser, Tracy A. 分享 收藏
Pharmacogenetics of Antiepileptic Drug Efficacy in Childhood Absence Epilepsy Glauser, Tracy A.; Holland, Katherine; O'Brien, Valerie P.; Keddache, Mehdi; Martin, Lisa J.; Clark, Peggy O.; Cnaan, Avital; Dlugos, Dennis; Hirtz, Deborah G.; Shinnar, Shlomo; Grabowski, Gregory 分享 收藏
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Long-term outcomes of generalized tonic-clonic seizures in a childhood absence epilepsy trial Shinnar, Shlomo; Cnaan, Avital; Hu, Fengming; Clark, Peggy; Dlugos, Dennis; Hirtz, Deborah G.; Masur, David; Mizrahi, Eli M.; Moshe, Solomon L.; Glauser, Tracy A. 分享 收藏
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Feasibility and Reproducibility of Echocardiographic Measures in Children with Muscular Dystrophies 肌营养不良儿童超声心动图测量的可行性和可重复性 Spurney, Christopher F.; McCaffrey, Francis M.; Cnaan, Avital; Morgenroth, Lauren P.; Ghelani, Sunil J.; Gordish-Dressman, Heather; Arrieta, Adrienne; Connolly, Anne M.; Lotze, Timothy E.; McDonald, Craig M.; Leshner, Robert T.; Clemens, Paula R. 分享 收藏
Genetic Modifiers of Ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study Bello, Luca; Kesari, Akanchha; Gordish-Dressman, Heather; Cnaan, Avital; Morgenroth, Lauren P.; Punetha, Jaya; Duong, Tina; Henricson, Erik K.; Pegoraro, Elena; McDonald, Craig M.; Hoffman, Eric P. 分享 收藏
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