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Tumor volume features predict survival outcomes for patients diagnosed with diffuse intrinsic pontine glioma 肿瘤体积特征可预测诊断为弥漫性内在性脑桥胶质瘤的患者的生存结局 Spencer, D'Andre; Bonner, Erin R.; Tor-Diez, Carlos; Liu, Xinyang; Bougher, Kristen; Prasad, Rachna; Gordish-Dressman, Heather; Eze, Augustine; Packer, Roger J.; Nazarian, Javad; Linguraru, Marius George; Bornhorst, Miriam 分享 收藏
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Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial Lawlor, Michael W.; Schoser, Benedikt; Margeta, Marta; Sewry, Caroline A.; Jones, Karra A.; Shieh, Perry B.; Kuntz, Nancy L.; Smith, Barbara K.; Dowling, James J.; Mueller-Felber, Wolfgang; Boennemann, Carsten G.; Seferian, Andreea M.; Blaschek, Astrid; Neuhaus, Sarah; Foley, A. Reghan; Saade, Dimah N.; Tsuchiya, Etsuko; Qasim, Ummulwara R.; Beatka, Margaret; Prom, Mariah J.; Ott, Emily; Danielson, Susan; Krakau, Paul; Kumar, Suresh N.; Meng, Hui; Vanden Avond, Mark; Wells, Clive; Gordish-Dressman, Heather; Beggs, Alan H.; Christensen, Sarah; Conner, Edward; James, Emma S.; Lee, Jun; Sadhu, Chanchal; Miller, Weston; Sepulveda, Bryan; Varfaj, Fatbardha; Prasad, Suyash; Rico, Salvador 分享 收藏
Findings from the Longitudinal CINRG Becker Natural History Study Clemens, Paula R.; Gordish-Dressman, Heather; Niizawa, Gabriela; Gorni, Ksenija; Guglieri, Michela; Connolly, Anne M.; Wicklund, Matthew; Bertorini, Tulio; Mah, Jean; Thangarajh, Mathula; Smith, Edward C.; Kuntz, Nancy L.; McDonald, Craig M.; Henricson, Erik; Upadhyayula, S.; Byrne, Barry; Manousakis, Georgios; Harper, Amy; Iannaccone, Susan; Dang, Utkarsh J. 分享 收藏
Ataluren Preserves Upper Limb Function in nmDMD Patients from Study 041, a Phase 3 Placebo-Controlled Trial, and the STRIDE Registry McDonald, Craig M.; Mercuri, Eugenio; Muntoni, Francesco; Gordish-Dressman, Heather; Morgenroth, Lauren P.; De Resende, Maria Bernadete Dutra; Zhou, Shuizhen; Neeharika, Mathukumalli Lakshmi; Haginoya, Kazuhiro; Ramos-Platt, Leigh; Williams, Paula; Penematsa, Vinay; Chou, Connie; Lin, Min; Johnson, Shelley; Koladicz, Karyn; Mastrandrea, Nicholas; Werner, Christian; Trifillis, Panayiota 分享 收藏
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Circulating tumor DNA sequencing provides comprehensive mutation profiling for pediatric central nervous system tumors Bonner, Erin R.; Harrington, Robin; Eze, Augustine; Bornhorst, Miriam; Kline, Cassie N.; Gordish-Dressman, Heather; Dawood, Adam; Das, Biswajit; Chen, Li; Pauly, Rini; Williams, P. Mickey; Karlovich, Chris; Peach, Amanda; Howell, D'andra; Doroshow, James; Kilburn, Lindsay; Packer, Roger J.; Mueller, Sabine; Nazarian, Javad 分享 收藏
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 分享 收藏
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Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy Heier, Christopher R.; Zhang, Aiping; Nguyen, Nhu Y.; Tully, Christopher B.; Panigrahi, Aswini; Gordish-Dressman, Heather; Pandey, Sachchida Nand; Guglieri, Michela; Ryan, Monique M.; Clemens, Paula R.; Thangarajh, Mathula; Webster, Richard; Smith, Edward C.; Connolly, Anne M.; McDonald, Craig M.; Karachunski, Peter; Tulinius, Mar; Harper, Amy; Mah, Jean K.; Fiorillo, Alyson A.; Chen, Yi-Wen 分享 收藏
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Protective effect of HLA-DQB1 alleles against alloimmunization in patients with sickle cell disease (vol 77, pg 35, 2016) Tatari-Calderone, Z.; Gordish-Dressman, H.; Fasano, R.; Riggs, M.; Fortier, C.; Campbell, A. D.; Charron, D.; Gordeuk, V. R.; Luban, N. L. C.; Vukmanovic, S.; Tamouza, R. 分享 收藏
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Suitability of external controls for drug evaluation in Duchenne muscular dystrophy Goemans, Nathalie; Signorovitch, James; Sajeev, Gautam; Yao, Zhiwen; Gordish-Dressman, Heather; McDonald, Craig M.; Vandenborne, Krista; Miller, Debra; Ward, Susan J.; Mercuri, Eugenio 分享 收藏
Genetic modifiers of respiratory function in Duchenne muscular dystrophy Duchenne肌营养不良症呼吸功能的遗传修饰 Bello, Luca; D'Angelo, Grazia; Villa, Matteo; Fusto, Aurora; Vianello, Sara; Merlo, Beatrice; Sabbatini, Daniele; Barp, Andrea; Gandossini, Sandra; Magri, Francesca; Comi, Giacomo P.; Pedemonte, Marina; Tacchetti, Paola; Lanzillotta, Valentina; Trucco, Federica; D'Amico, Adele; Bertini, Enrico; Astrea, Guja; Politano, Luisa; Masson, Riccardo; Baranello, Giovanni; Albamonte, Emilio; De Mattia, Elisa; Rao, Fabrizio; Sansone, Valeria A.; Previtali, Stefano; Messina, Sonia; Vita, Gian Luca; Berardinelli, Angela; Mongini, Tiziana; Pini, Antonella; Pane, Marika; Mercuri, Eugenio; Vianello, Andrea; Bruno, Claudio; Hoffman, Eric P.; Morgenroth, Lauren; Gordish-Dressman, Heather; McDonald, Craig M.; Pegoraro, Elena 分享 收藏
TGF-β-driven muscle degeneration and failed regeneration underlie disease onset in a DMD mouse model Mazala, Davi A. G.; Novak, James S.; Hogarth, Marshall W.; Nearing, Marie; Adusumalli, Prabhat; Tully, Christopher B.; Habib, Nayab F.; Gordish-Dressman, Heather; Chen, Yi-Wen; Jaiswal, Jyoti K.; Partridge, Terence A. 分享 收藏
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A decade of optimizing drug development for rare neuromuscular disorders through TACT Wagner, Kathryn R.; De Luca, Annamaria; Caizergues, Didier; Dowling, James; Goemans, Nathalie; Gordish-Dressman, Heather; Grounds, Miranda D.; Kelly, Michael; Mayhew, Anna; McNally, Elizabeth M.; Zoetis, Tracey; Lee, Joanne; Turner, Cathy; Wells, Dominic J.; Csimma, Cristina; Straub, Volker 分享 收藏
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