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Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy An Exploratory Analysis From the EMBARK Randomized Clinical Trial Vandenborne, Krista; Walter, Glenn A.; Straub, Volker; Willcocks, Rebecca J.; Forbes, Sean C.; Mercuri, Eugenio M.; Muntoni, Francesco; Ding, Kai; Ennamuri, Sravya; Reid, Carol; Murphy, Alexander P.; Manfrini, Marianna; Mendell, Jerry R.; Elkins, Jacob S.; Rodino-Klapac, Louise R. Share Save
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial Mendell, Jerry R.; Muntoni, Francesco; McDonald, Craig M.; Mercuri, Eugenio M.; Ciafaloni, Emma; Komaki, Hirofumi; Leon-Astudillo, Carmen; Nascimento, Andres; Proud, Crystal; Schara-Schmidt, Ulrike; Veerapandiyan, Aravindhan; Zaidman, Craig M.; Guridi, Maitea; Murphy, Alexander P.; Reid, Carol; Wandel, Christoph; Asher, Damon R.; Darton, Eddie; Mason, Stefanie; Potter, Rachael A.; Singh, Teji; Zhang, Wenfei; Fontoura, Paulo; Elkins, Jacob S.; Rodino-Klapac, Louise R. Share Save
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. Share Save
Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy Mendell, Jerry R.; Proud, Crystal; Zaidman, Craig M.; Mason, Stefanie; Darton, Eddie; Wang, Shufang; Wandel, Christoph; Murphy, Alexander P.; Mercuri, Eugenio; Muntoni, Francesco; Mcdonald, Craig M. Share Save
Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results Mendell, Jerry R.; Pozsgai, Eric R.; Lewis, Sarah; Griffin, Danielle A.; Lowes, Linda P.; Alfano, Lindsay N.; Lehman, Kelly J.; Church, Kathleen; Reash, Natalie F.; Iammarino, Megan A.; Sabo, Brenna; Potter, Rachael; Neuhaus, Sarah; Li, Xiaoxi; Stevenson, Herb; Rodino-Klapac, Louise R. Share Save
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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged=4 to <8?Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR) Zaidman, Craig M.; Proud, Crystal M.; Mcdonald, Craig M.; Lehman, Kelly J.; Goedeker, Natalie L.; Mason, Stefanie; Murphy, Alexander P.; Guridi, Maitea; Wang, Shufang; Reid, Carol; Darton, Eddie; Wandel, Christoph; Lewis, Sarah; Malhotra, Jyoti; Griffin, Danielle A.; Potter, Rachael A.; Rodino-Klapac, Louise R.; Mendell, Jerry R. Share Save
A Phase 2 Clinical Trial Evaluating the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Patients with Duchenne Muscular Dystrophy (DMD) Mendell, Jerry; Shieh, Perry; Sahenk, Zarife; Lehman, Kelly; Lowes, Linda; Reash, Natalie; Iammarino, Megan; Alfano, Lindsay; Sabo, Brenna; Woods, Jeremy; Skura, Christy; Mao, Howard; Staudt, Loretta; Potter, Rachael; Griffin, Danielle; Lewis, Sarah; Wang, Shufang; Singh, Tejdip; Rodino-Klapac, Louise Share Save
Water T2 could predict functional decline in patients with dysferlinopathy Moore, Ursula; Araujo, Ericky Caldas de Almeida; Reyngoudt, Harmen; Gordish-Dressman, Heather; Smith, Fiona E.; Wilson, Ian; James, Meredith; Mayhew, Anna; Rufibach, Laura; Day, John W.; Jones, Kristi J.; Bharucha-Goebel, Diana X.; Salort-Campana, Emmanuelle; Pestronk, Alan; Walter, Maggie C.; Paradas, Carmen; Stojkovic, Tanya; Mori-Yoshimura, Madoka; Bravver, Elena; Pegoraro, Elena; Mendell, Jerry R.; Bushby, Kate; Blamire, Andrew M.; Straub, Volker; Carlier, Pierre G.; Diaz-Manera, Jordi Share Save
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial McDonald, Craig M.; Shieh, Perry B.; Abdel-Hamid, Hoda Z.; Connolly, Anne M.; Ciafaloni, Emma; Wagner, Kathryn R.; Goemans, Nathalie; Mercuri, Eugenio; Khan, Navid; Koenig, Erica; Malhotra, Jyoti; Zhang, Wenfei; Han, Baoguang; Mendell, Jerry R. Share Save
Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale Jacobs, Marni B.; James, Meredith K.; Lowes, Linda P.; Alfano, Lindsay N.; Eagle, Michelle; Muni Lofra, Robert; Moore, Ursula; Feng, Jia; Rufibach, Laura E.; Rose, Kristy; Duong, Tina; Bello, Luca; Pedrosa-Hernandez, Irene; Holsten, Scott; Sakamoto, Chikako; Canal, Aurelie; Sanchez-Aguilera Praxedes, Nieves; Thiele, Simone; Siener, Catherine; Vandevelde, Bruno; DeWolf, Brittney; Maron, Elke; Guglieri, Michela; Hogrel, Jean-Yves; Blamire, Andrew M.; Carlier, Pierre G.; Spuler, Simone; Day, John W.; Jones, Kristi J.; Bharucha-Goebel, Diana X.; Salort-Campana, Emmanuelle; Pestronk, Alan; Walter, Maggie C.; Paradas, Carmen; Stojkovic, Tanya; Mori-Yoshimura, Madoka; Bravver, Elena; Diaz-Manera, Jordi; Pegoraro, Elena; Mendell, Jerry R.; Mayhew, Anna G.; Straub, Volker Share Save
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Natural History of Steroid-Treated Young Boys With Duchenne Muscular Dystrophy Using the NSAA, 100m, and Timed Functional Tests Miller, Natalie F.; Alfano, Lindsay N.; Iammarino, Megan A.; Connolly, Anne M.; Moore-Clingenpeel, Melissa; Powers, Brenna R.; Tsao, Chang-Yong; Waldrop, Megan A.; Flanigan, Kevin M.; Mendell, Jerry R.; Lowes, Linda P. Share Save
Duchenne and Becker muscular dystrophy carriers: Evidence of cardiomyopathy by exercise and cardiac MRI testing Mah, May Ling; Cripe, Linda; Slawinski, Michelle K.; Al-Zaidy, Samiah A.; Camino, Eric; Lehman, Kelly J.; Jackson, Jamie L.; Iammarino, Megan; Miller, Natalie; Mendell, Jerry R.; Hor, Kan N. Share Save
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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. Share Save
Eteplirsen treatment for Duchenne muscular dystrophy Exon skipping and dystrophin production Charleston, Jay S.; Schnell, Frederick J.; Dworzak, Johannes; Donoghue, Cas; Lewis, Sarah; Chen, Lei; Young, G. David; Milici, Anthony J.; Voss, Jon; DeAlwis, Uditha; Wentworth, Bruce; Rodino-Klapac, Louise R.; Sahenk, Zarife; Frank, Diane; Mendell, Jerry R. Share Save
Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials Diaz-Manera, Jordi; Fernandez-Torron, Roberto; LLauger, Jaume; James, Meredith K.; Mayhew, Anna; Smith, Fiona E.; Moore, Ursula R.; Blamire, Andrew M.; Carlier, Pierre G.; Rufibach, Laura; Mittal, Plavi; Eagle, Michelle; Jacobs, Marni; Hodgson, Tim; Wallace, Dorothy; Ward, Louise; Smith, Mark; Stramare, Roberto; Rampado, Alessandro; Sato, Noriko; Tamaru, Takeshi; Harwick, Bruce; Gala, Susana Rico; Turk, Suna; Coppenrath, Eva M.; Foster, Glenn; Bendahan, David; Le Fur, Yann; Fricke, Stanley T.; Otero, Hansel; Foster, Sheryl L.; Peduto, Anthony; Sawyer, Anne Marie; Hilsden, Heather; Lochmuller, 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; Bello, Luca; Semplicini, Claudio; Pegoraro, Elena; Mendell, Jerry R.; Bushby, Kate; Straub, Volker Share Save
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