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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. Among authors: mccall j. 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.
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: mccall jm. 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.
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function.
Hoffman EP, Schwartz BD, Mengle-Gaw LJ, Smith EC, Castro D, Mah JK, McDonald CM, Kuntz NL, Finkel RS, Guglieri M, Bushby K, Tulinius M, Nevo Y, Ryan MM, Webster R, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Siener C, Jaros M, Shale P, McCall JM, Nagaraju K, van den Anker J, Conklin LS, Cnaan A, Gordish-Dressman H, Damsker JM, Clemens PR; Cooperative International Neuromuscular Research Group. Hoffman EP, et al. Among authors: mccall jm. Neurology. 2019 Sep 24;93(13):e1312-e1323. doi: 10.1212/WNL.0000000000008168. Epub 2019 Aug 26. Neurology. 2019. PMID: 31451516 Free PMC article. Clinical Trial.
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: mccall jm. 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.
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.
Conklin LS, Damsker JM, Hoffman EP, Jusko WJ, Mavroudis PD, Schwartz BD, Mengle-Gaw LJ, Smith EC, Mah JK, Guglieri M, Nevo Y, Kuntz N, McDonald CM, Tulinius M, Ryan MM, Webster R, Castro D, Finkel RS, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, McCall JM, Hathout Y, Nagaraju K, van den Anker J, Ward LM, Ahmet A, Cornish MR, Clemens PR. Conklin LS, et al. Among authors: mccall jm. Pharmacol Res. 2018 Oct;136:140-150. doi: 10.1016/j.phrs.2018.09.007. Epub 2018 Sep 13. Pharmacol Res. 2018. PMID: 30219580 Free PMC article. Clinical Trial.
Δ-9,11 modification of glucocorticoids dissociates nuclear factor-κB inhibitory efficacy from glucocorticoid response element-associated side effects.
Baudy AR, Reeves EK, Damsker JM, Heier C, Garvin LM, Dillingham BC, McCall J, Rayavarapu S, Wang Z, Vandermeulen JH, Sali A, Jahnke V, Duguez S, DuBois D, Rose MC, Nagaraju K, Hoffman EP. Baudy AR, et al. Among authors: mccall j. J Pharmacol Exp Ther. 2012 Oct;343(1):225-32. doi: 10.1124/jpet.112.194340. Epub 2012 Jun 28. J Pharmacol Exp Ther. 2012. PMID: 22743576 Free PMC article.
VBP15, a glucocorticoid analogue, is effective at reducing allergic lung inflammation in mice.
Damsker JM, Dillingham BC, Rose MC, Balsley MA, Heier CR, Watson AM, Stemmy EJ, Jurjus RA, Huynh T, Tatem K, Uaesoontrachoon K, Berry DM, Benton AS, Freishtat RJ, Hoffman EP, McCall JM, Gordish-Dressman H, Constant SL, Reeves EK, Nagaraju K. Damsker JM, et al. Among authors: mccall jm. PLoS One. 2013 May 7;8(5):e63871. doi: 10.1371/journal.pone.0063871. Print 2013. PLoS One. 2013. PMID: 23667681 Free PMC article.
834 results