Relevant publications

Future clinical trial design and innovative end points (Biomarkers and Imaging) for DMD and other Neuromuscular diseases workshop report 2019

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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

Edward C. Smith, Laurie S. Conklin2,3, Eric P. Hoffman, Paula R. Clemens, Jean K. Mah, Richard S. Finkel, Michela Guglieri, Mar Tulinius, Yoram Nevo, Monique
M. Ryan, Richard Webster, Diana Castro, Nancy L. Kuntz, Laurie Kerchner,Lauren P. Morgenroth, Adrienne Arrieta, Maya Shimony, Mark Jaros, Phil Shale, Heather Gordish-Dressman, Laura Hagerty, Utkarsh J. Dang, Jesse M. Damsker, Benjamin D. Schwartz, Laurel J. Mengle-Gaw, Craig M. McDonaldID, the CINRG VBP15 and DNHS Investigators

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Disruption of a key ligand-H-bond network drives dissociative properties in vamorolone for Duchenne muscular dystrophy treatment

Xu Liu, Yashuo Wang, Jennifer S. Gutierrez, Jesse M. Damsker, Kanneboyina Nagarajub, Eric P. Hoffman,and Eric A. Ortlund

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SHOWCASING EU FUNDED PROJECTS DEVELOPING THERAPIES FOR RARE DISEASES

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Vamorolone trial in Duchenne muscular dystrophy shows dose related improvement of muscle function

E. P. Hoffman, PhD, B. D. Schwartz, MD, L. J. Mengle-Gaw, PhD, E. C. Smith, MD, D. Castro, MD, J. K. Mah, MD, C. M. McDonald, MD, N. L. Kuntz, MD, R.S. Finkel, MD, M.Guglieri, MD, K.Bushby, MD, M.Tulinius, MD, Y. Nevo, MD, M.M. Ryan, MD, R. Webster, MD, A. L. Smith, MS, L.P. Morgenroth, MS, A.Arrieta, MS, M. Shimony, MPH, C.Siener, PT, M. Jaros, PhD, P.Shale, PhD, J.M. McCall, PhD, A.Nagaraju, PhD, DVM, J. van den Anker, MD, PhD, L. S. Conklin, MD, A.Cnaan, PhD, H. Gordish-Dressman, PhD, J. M. Damsker, PhD, and P. R. Clemens, MD, and the CINRG group.
Neurology® 2019;93:1-12. doi:10.1212/WNL.0000000000008168

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Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug

Conklin L, Damsker JM, Hoffman EP, Juskod WP, Panteleimon MD, Schwartze BD, Mengle-Gawe LJ, Smith EC, Mahg JK, Guglieri M, Nevoi Y, Kuntz N, McDonald CM, Tulinius M, Ryan M, Webster R, Castro D, Finkel RS, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, McCall JM, Hathout Y, Nagaraju K, Anker J, Ward LM, Ahmet A, Cornish MR, Clemens PR.    https://doi.org/10.1016/j.phrs.2018.09.007

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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.   https://doi.org/10.1152/physiolgenomics.00134.2017

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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

Hoffman EP, Riddle V, Siegler MA, Dickerson D, Backonja M, Kramer WG, Nagaraju K, Gordish-Dressmen H, Damsker JM, McCall JM.Hoffman,E.P.,Steroids(2018),htps://doi.org/10.1016/j.steroids.2018.02.010

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Quantifying fat replacement of muscle by quantitative MRI in muscular dystrophy

J. Burakiewicz , C. Sinclair, D. Fischer, G. Walter, H. Kan, K. Hollingsworth. J Neurol (2017) 264:2053–2067 DOI 10.1007/s00415-017-8547-3

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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.

Damsker 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.

Garvin 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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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.
EMBO Mol Med. 2013 Oct;5(10):1569-85

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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.
PLoS One. 2013 May 7;8(5):e63871

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VBP15: preclinical characterisation of a novel anti-inflammatory delta 9,11 steroid.

Reeves EK, Hoffman EP, Nagaraju K, Damsker JM, McCall JM.
Bioorg Med Chem. 2013 Apr 15;21(8):2241-9.

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Large-scale serum protein biomarker discovery in Duchenne muscular dystophy

Hathout Y, Brody E, Clemens PR, et al.  Proceedings of the National Academy of Sciences of the United States of America. 2015;112(23):7153-7158. doi:10.1073/pnas.1507719112.

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