Publications
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial
Guglieri 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
Mah 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
Liu 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
Smith 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
Li 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
Mavroudis PD1, van den Anker J2,3, Conklin LS2,3, Damsker JM2, Hoffman EP2,4, Nagaraju K2,4, Clemens PR5, Jusko WJ1.
Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy
Heier 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.
Vamorolone, a dissociative steroidal compound, reduces collagen antibody-induced joint damage and inflammation when administered after disease onset
Damsker 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.
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement in muscle function
Hoffman 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.
Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone
Fiorillo 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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