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Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial
PubsGuglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans N, Haberlova J, Straub V, Mengle-Gaw LJ, Schwartz BD, Harper AD, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster R, McMillan HJ, Kuntz NL, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez-Padilla JJ, Nascimento-Osorio A, Niks EH, de Groot IJM, Katsalouli M, James MK, van den Anker J, Damsker JM, Ahmet A, Ward LM, Jaros M, Shale P, Dang UJ, Hoffman EP. JAMA Neurol. 2022 Oct 1;79(10):1005-1014. doi: 10.1001/jamaneurol.2022.2480. PMID: 36036925; PMCID: PMC9425287.
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Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial
PubsMah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ; NorthStar UK Network and CINRG DNHS Investigators. JAMA Netw Open. 2022 Jan 4;5(1):e2144178. doi: 10.1001/jamanetworkopen.2021.44178. PMID: 35076703; PMCID: PMC8790668.
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Disruption of a key ligand-H-bond network drives dissociative properties in vamorolone for Duchenne muscular dystrophy treatment
PubsLiu X, Wang Y, Gutierrez JS, Damsker JM, Nagaraju K, Hoffman EP, Ortlund EA. Proc Natl Acad Sci U S A. 2020 Sep 29;117(39):24285-24293. doi: 10.1073/pnas.2006890117. Epub 2020 Sep 11. PMID: 32917814; PMCID: PMC7533876.
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Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study
PubsSmith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM; CINRG VBP15 and DNHS Investigators. PLoS Med. 2020 Sep 21;17(9):e1003222. doi: 10.1371/journal.pmed.1003222. PMID: 32956407; PMCID: PMC7505441.
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Exposure-Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy
PubsLi X, Conklin LS, van den Anker J, Hoffman EP, Clemens PR, Jusko WJ. J Clin Pharmacol. 2020 Oct;60(10):1385-1396. doi: 10.1002/jcph.1632. Epub 2020 May 20. PMID: 32434278; PMCID: PMC7494537.
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Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy
PubsMavroudis PD1, van den Anker J2,3, Conklin LS2,3, Damsker JM2, Hoffman EP2,4, Nagaraju K2,4, Clemens PR5, Jusko WJ1.
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Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy
PubsHeier CR1,2, Yu Q2, Fiorillo AA3,2, Tully CB2, Tucker A2, Mazala DA2, Uaesoontrachoon K4, Srinivassane S4, Damsker JM5, Hoffman EP4,5,6, Nagaraju K4,5,6, Spurney CF3,2,7.
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Vamorolone, a dissociative steroidal compound, reduces collagen antibody-induced joint damage and inflammation when administered after disease onset
PubsDamsker JM1, Cornish MR2, Kanneboyina P2, Kanneboyina I2, Yu Q3, Lipson R2, Phadke A3, Knoblach SM3, Panchapakesan K3, Morales M4, Fiorillo AA3, Partridge T3, Nagaraju K5,6,7.
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Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement in muscle function
PubsHoffman EP1, Schwartz BD2, Mengle-Gaw LJ2, Smith EC2, Castro D2, Mah JK2, McDonald CM2, Kuntz NL2, Finkel RS2, Guglieri M2, Bushby K2, Tulinius M2, Nevo Y2, Ryan MM2, Webster R2, Smith AL2, Morgenroth LP2, Arrieta A2, Shimony M2, Siener C2, Jaros M2, Shale P2, McCall JM2, Nagaraju K2, van den Anker J2, Conklin LS2, Cnaan A2, Gordish-Dressman H2, Damsker JM2, Clemens PR2; Cooperative International Neuromuscular Research Group.
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Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone
PubsFiorillo AA, Tully CB, Damsker JM, Nagaraju K, Hoffman EP, Heier CR.
Physiol Genomics. 2018 Sep 1;50(9):735-745. PMID: 29883261
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Vamorolone treatment improves skeletal muscle outcome in a critical illness myopathy rat model.
PubsAkkad H, Cacciani N, Llano-Diez M, Corpeno Kalamgi R, Tchkonia T, Kirkland JL, Larsson L.
Acta Physiol (Oxf). 2018 Aug 18:e13172. PMID: 30120816
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Membrane Stabilization by Modified Steroid Offers a Potential Therapy for Muscular Dystrophy Due to Dysferlin Deficit
PubsSreetama SC, Chandra G, Van der Meulen JH, Ahmad MM, Suzuki P, Bhuvanendran S, Nagaraju K, Hoffman EP, Jaiswal JK.
Mol Ther. 2018 Sep 5;26(9):2231-2242. PMID: 30166241
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Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug
PubsConklin 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.
Pharmacol Res. 2018 Sep 13;136:140-150.
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Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes
PubsHoffman EP, Riddle V, Siegler MA, Dickerson D, Backonia M, Kramer WG, et al. Steroids. 2018; 134: 43-52
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Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone
PubsFiorillo AA, Tully CB, Damsker JM, Nagaraju K, Hoffman EP, Heier CR. Physiol Genomics. 2018.
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VBP15, a novel dissociative steroid compound, reduces NFκB-induced expression of inflammatory cytokines in vitro and symptoms of murine trinitrobenzene sulfonic acid-induced colitis.
PubsDamsker JM, Conklin LS, Sadri S, Dillingham BC, Panchapakesan K, Heier CR, McCall JM, Sandler AD.
Inflamm Res. 2016 Sep;65(9):737-43. doi: 10.1007/s00011-016-0956-8. Epub 2016 Jun 3.
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A novel dissociative steroid VBP15 reduces MUC5AC gene expression in airway epithelial cells but lacks the GRE mediated transcriptional properties of dexamethasone.
PubsGarvin LM1, Chen Y2, Damsker JM3, Rose MC4.
Pulm Pharmacol Ther. 2016 Jun;38:17-26. doi: 10.1016/j.pupt.2016.04.004. Epub 2016 Apr 29.
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Pharmacologically-induced mitotic synchrony in airway epithelial cells as a mechanism of action of anti-inflammatory drugs.
PubsFreishtat RJ, Nino G, Tsegaye Y, Alcala SE, Benton AS, Watson AM, Reeves EK, Haider SK, Damsker JM.
Respir Res. 2015 Oct 29;16:132. doi: 10.1186/s12931-015-0293-4.
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Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy.
PubsDadgar S, Wang Z, Johnston H, Kesari A, Nagaraju K, Chen YW, Hill DA, Partridge TA, Giri M, Freishtat RJ, Nazarian J, Xuan J, Wang Y, Hoffman EP.
J Cell Biol. 2014 Oct 13;207(1):139-58. doi: 10.1083/jcb.201402079.
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VBP15, a novel anti-inflammatory, is effective at reducing the severity of murine experimental autoimmune encephalomyelitis.
PubsDillingham BC, Knoblach SM, Many GM, Harmon BT, Mullen AM, Heier CR, Bello L, McCall JM, Hoffman EP, Connor EM, Nagaraju K, Reeves EK, Damsker JM.
Cell Mol Neurobiol. 2015 Apr;35(3):377-87. doi: 10.1007/s10571-014-0133-y. Epub 2014 Nov 13.
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