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Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy Verhaart, Ingrid E. C.; Cappellari, Ornella; Tanganyika-de Winter, Christa L.; Plomp, Jaap J.; Nnorom, Sofia; Wells, Kim E.; Hildyard, John C. W.; Bull, David; Aartsma-Rus, Annemieke; Wells, Dominic J. 分享 收藏
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Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT) Willmann, Raffaella; Lee, Joanne; Turner, Cathy; Nagaraju, Kanneboyina; Aartsma-Rus, Annemieke; Wells, Dominic J.; Wagner, Kathryn R.; Csimma, Cristina; Straub, Volker; Grounds, Miranda D.; De Luca, Annamaria 分享 收藏
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
Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment Cmah-肌营养不良蛋白缺陷型mdx小鼠表现出加速的心脏表型,在肽-PMO外显子跳跃处理后得到改善 Betts, Corinne A.; McClorey, Graham; Healicon, Richard; Hammond, Suzan M.; Manzano, Raquel; Muses, Sofia; Ball, Vicky; Godfrey, Caroline; Merritt, Thomas M.; van Westering, Tirsa; O'Donovan, Liz; Wells, Kim E.; Gait, Michael J.; Wells, Dominic J.; Tyler, Damian; Wood, Matthew J. 分享 收藏
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Three-Dimensional Human iPSC-Derived Artificial Skeletal Muscles Model Muscular Dystrophies and Enable Multilineage Tissue Engineering Maffioletti, Sara Martina; Sarcar, Shilpita; Henderson, Alexander B. H.; Mannhardt, Ingra; Pinton, Luca; Moyle, Louise Anne; Steele-Stallard, Heather; Cappellari, Ornella; Wells, Kim E.; Ferrari, Giulia; Mitchell, Jamie S.; Tyzack, Giulia E.; Kotiadis, Vassilios N.; Khedr, Moustafa; Ragazzi, Martina; Wang, Weixin; Duchen, Michael R.; Patani, Rickie; Zammit, Peter S.; Wells, Dominic J.; Eschenhagen, Thomas; Tedesco, Francesco Saverio 分享 收藏
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Growth differentiation factor-15 is associated with muscle mass in chronic obstructive pulmonary disease and promotes muscle wasting in vivo Patel, Mehul S.; Lee, Jen; Baz, Manuel; Wells, Claire E.; Bloch, Susannah; Lewis, Amy; Donaldson, Anna V.; Garfield, Benjamin E.; Hopkinson, Nicholas S.; Natanek, Amanda; Man, William D-C; Wells, Dominic J.; Baker, Emma H.; Polkey, Michael I.; Kemp, Paul R. 分享 收藏
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Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy Betts, Corinne A.; Saleh, Amer F.; Carr, Carolyn A.; Muses, Sofia; Wells, Kim E.; Hammond, Suzan M.; Godfrey, Caroline; McClorey, Graham; Woffindale, Caroline; Clarke, Kieran; Wells, Dominic J.; Gait, Michael J.; Wood, Matthew J. A. 分享 收藏
How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse Godfrey, Caroline; Muses, Sofia; McClorey, Graham; Wells, Kim E.; Coursindel, Thibault; Terry, Rebecca L.; Betts, Corinne; Hammond, Suzan; O'Donovan, Liz; Hildyard, John; El Andaloussi, Samir; Gait, Michael J.; Wood, Matthew J.; Wells, Dominic J. 分享 收藏
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development Heslop, Emma; Csimma, Cristina; Straub, Volker; McCall, John; Nagaraju, Kanneboyina; Wagner, Kathryn R.; Caizergues, Didier; Korinthenberg, Rudolf; Flanigan, Kevin M.; Kaufmann, Petra; McNeil, Elizabeth; Mendell, Jerry; Hesterlee, Sharon; Wells, Dominic J. 分享 收藏