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Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy.
Hoffman EP, Reeves E, Damsker J, Nagaraju K, McCall JM, Connor EM, Bushby K. Hoffman EP, et al. Among authors: connor em. Phys Med Rehabil Clin N Am. 2012 Nov;23(4):821-8. doi: 10.1016/j.pmr.2012.08.003. Phys Med Rehabil Clin N Am. 2012. PMID: 23137739 Free PMC article. Review.
VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects.
Heier CR, Damsker JM, Yu Q, Dillingham BC, Huynh T, Van der Meulen JH, Sali A, Miller BK, Phadke A, Scheffer L, Quinn J, Tatem K, Jordan S, Dadgar S, Rodriguez OC, Albanese C, Calhoun M, Gordish-Dressman H, Jaiswal JK, Connor EM, McCall JM, Hoffman EP, Reeves EK, Nagaraju K. Heier CR, et al. Among authors: connor em. EMBO Mol Med. 2013 Oct;5(10):1569-85. doi: 10.1002/emmm.201302621. Epub 2013 Sep 9. EMBO Mol Med. 2013. PMID: 24014378 Free PMC article.
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development.
Heslop E, Csimma C, Straub V, McCall J, Nagaraju K, Wagner KR, Caizergues D, Korinthenberg R, Flanigan KM, Kaufmann P, McNeil E, Mendell J, Hesterlee S, Wells DJ, Bushby K; TACT. Heslop E, et al. Orphanet J Rare Dis. 2015 Apr 23;10:49. doi: 10.1186/s13023-015-0258-1. Orphanet J Rare Dis. 2015. PMID: 25902795 Free PMC article. Review.
Meeting the demand for pediatric clinical trials.
Connor EM, Smoyer WE, Davis JM, Zajicek A, Ulrich L, Purucker M, Hirschfeld S. Connor EM, et al. Sci Transl Med. 2014 Mar 12;6(227):227fs11. doi: 10.1126/scitranslmed.3008043. Sci Transl Med. 2014. PMID: 24622511 Free PMC article.
89 results