VISION-DMD - Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy

Project Information

VISION DMD

Grant agreement ID: 667078

DOI 10.3030/667078

Project closed

EC signature date 9 December 2015

Start date 1 January 2016

End date 31 December 2021

Funded under

• SOCIETAL CHALLENGES - Health, demographic change and well-being

Total cost € 16 858 748,72

EU contribution € 6 000 000,00

Coordinated by

UNIVERSITY OF NEWCASTLE UPON TYNE
United Kingdom

Deliverables

Websites, patent fillings, videos etc. (1)

Project Website

Develop and publish project website including public summaries of activity

Demonstrators, pilots, prototypes (1)

VISION DMD Workshop Series

Completion of workshop series

Publications

Peer reviewed articles (7)

Pubertal induction in adolescents with DMD is associated with high satisfaction, gonadotropin release and increased muscle contractile surface area

Author(s): Claire L Wood Kieren G Hollingsworth, Eric Hughes, Sadhanandham Punniyakodi, Robert Muni-Lofra, Anna Mayhew, Rod T Mitchell, Michela Guglieri, Timothy D Cheetham, and Volker Straub
Published in: European Journal of Endocrinology, Issue 2021;184(1), 2021, Page(s) 67-79., ISSN 1479-683X
Publisher: European Society of Endocrinology
DOI: 10.1530/eje-20-0709

Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes

Author(s): Eric P. Hoffman, Valerie Riddle, Maxime A. Siegler, Daniel Dickerson, Miroslav Backonja, William G. Kramer, Kanneboyina Nagaraju, Heather Gordish-Dressman, Jesse M. Damsker, John M. McCall
Published in: Steroids, 2018, ISSN 0039-128X
Publisher: Elsevier BV
DOI: 10.1016/j.steroids.2018.02.010

Quantifying fat replacement of muscle by quantitative MRI in muscular dystrophy

Author(s): Jedrzej Burakiewicz, Christopher D. J. Sinclair, Dirk Fischer, Glenn A. Walter, Hermien E. Kan, Kieren G. Hollingsworth
Published in: Journal of Neurology, Issue 264/10, 2017, Page(s) 2053-2067, ISSN 0340-5354
Publisher: Dr. Dietrich Steinkopff Verlag
DOI: 10.1007/s00415-017-8547-3

Vision DMD: Vamorolone (VBP15) drug development program for Duchenne muscular dystrophy

Author(s): M. Guglieri, P. Clemens, A. Cnaan, J. Damsker, A. Arrieta, L. Morgenroth, R. Davis, C. Olsen, R. Head, K. Nagaraju, Y. Hathout, J. Haberlova, D. Athanasiou, E. Vroom, K. Bushby, E. Hoffman
Published in: European Journal of Paediatric Neurology, Issue 21, 2017, Page(s) e238, ISSN 1090-3798
Publisher: W. B. Saunders Co., Ltd.
DOI: 10.1016/j.ejpn.2017.04.1270

New therapies in neuromuscular disorders in childhood Nové možnosti léčby vrozených neuromuskulárních onemocnění v dětském věku

Author(s): MUDr. Jana Haberlová, Ph.D.
Published in: Neurology for Practice, Issue bimonthly, 2018, Page(s) 2018; 19(2): 108–113, ISSN 1213-1814
Publisher: NEUROLOGIE PRO PRAXI

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

Author(s): Eric P. Hoffman, Benjamin D. Schwartz, Laurel J. Mengle-Gaw, Edward C. Smith, Diana Castro, Jean K. Mah, Craig M. McDonald, Nancy L. Kuntz, Richard S. Finkel, Michela Guglieri, Katharine Bushby, Mar Tulinius, Yoram Nevo, Monique M. Ryan, Richard Webster, Andrea L. Smith, Lauren P. Morgenroth, Adrienne Arrieta, Maya Shimony, Catherine Siener, Mark Jaros, Phil Shale, John M. McCall, Kanneboyina Naga
Published in: Neurology, 2019, Page(s) 10.1212/WNL.0000000000008168, ISSN 0028-3878
Publisher: Lippincott Williams & Wilkins Ltd.
DOI: 10.1212/wnl.0000000000008168

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

Author(s): Laurie S. Conklin, Jesse M. Damsker, Eric P. Hoffman, William J. Jusko, Panteleimon D. Mavroudis, Benjamin D. Schwartz, Laurel J. Mengle-Gaw, Edward C. Smith, Jean K. Mah, Michela Guglieri, Yoram Nevo, Nancy Kuntz, Craig M. McDonald, Mar Tulinius, Monique M. Ryan, Richard Webster, Diana Castro, Richard S. Finkel, Andrea L. Smith, Lauren P. Morgenroth, Adrienne Arrieta, Maya Shimony, Mark Jaros, Ph
Published in: Pharmacological Research, Issue 136, 2018, Page(s) 140-150, ISSN 1043-6618
Publisher: Academic Press
DOI: 10.1016/j.phrs.2018.09.007

Other (2)

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

Author(s): Olsen, Christina; Head, Ritchie; Knerova, Jane; Larkindale, Jane; Ward, Susan; Spitali, Pietro; Kan, Hermien
Published in: Issue 2, 2021
Publisher: Zenodo
DOI: 10.5281/zenodo.5668576

RETURNING INDIVIDUAL CLINICAL TRIAL DATA BACK TO PARTICIPANTS

Author(s): Dr Laurie Conklin; Dr Holly Peay; Gaglianone, Suzanne; Suzie-Ann Bakker; Vroom, Elizabeth; Dr Eric Hoffman; Olsen, Christina
Published in: Issue 3, 2021
Publisher: Zenodo
DOI: 10.5281/zenodo.4836583