VISION-DMD

Designed to ensure a timely & cost-effective drug development for Duchenne muscular dystrophy

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    • Summary
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  • About DMD
    • What is DMD?
    • Living with Duchenne
    • Current treatment options
    • Duchenne boy’s stories
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    • Standards of Care for Duchenne muscular dystrophy
  • Study Information
    • What is a clinical study?
    • Phase 2a clinical trial information
    • Phase 2a preliminary study results
    • Phase 2b clinical trial information
    • Phase 2b Preliminary Results
    • MRI – Magnetic Resonance Imaging
  • Drug Development
    • Development of Vamorolone
    • Vamorolone development programme
  • Innovations
    • Return of patient data
    • Bone Health Outcomes
    • Biomarkers
    • Vamorolone study APP for boys
    • Patient Group Funders
    • What is Venture Philanthropy?
  • Resources
    • Relevant publications
      • VISION-DMD INFORMATION APP OVERVIEW
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      • Workshop series: Standards of Care
      • Workshop series: Biomarkers and Imaging
    • Webinars and presentations
    • Press Releases
    • VISION-DMD project Interviews
      • Collaborating for progress on rare diseases: an interview with Dr. Daria Julkowska, Scientific Coordinator of the European Joint Program on Rare Diseases.
      • Returning vamorolone study data to patient caregivers. What, when and how clinical trial data is returned to study participants. An interview with Dr. Laurie Conklin
      • An Interview with Andrea D’Alessandro – Clinical research project manager at TRiNDS
      • Interview with Jana Haberlová – Paediatric Neurologist at University Hospital Motol
      • Interview with Alex Johnson: a parent and patient foundation funder
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Relevant publications

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

Link to report

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

Link to article

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

Link to article

SHOWCASING EU FUNDED PROJECTS DEVELOPING THERAPIES FOR RARE DISEASES

Link to article

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

Link to article

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

Link to article

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

Link to full article

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

Link to full article

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

Link to full publication

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.

Link to full article:

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.

Link to full article:

VBP15, a novel anti-inflammatory, is effective at reducing the severity of murine experimental autoimmune encephalomyelitis.

Dillingham 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.

Link to full article

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

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

Link to full article:

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.

Link to abstract:

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.

Link to article

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This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement number 667078.

To find out more about the project contact us at [email protected]

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