VISION-DMD - Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy | VISION DMD | Project | Results | H2020 | CORDIS

Livrables

Develop and publish project website including public summaries of activity

Completion of workshop series

Publications

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

Auteurs: 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
Publié dans: European Journal of Endocrinology, Numéro 2021;184(1), 2021, Page(s) 67-79., ISSN 1479-683X
Éditeur: 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

Auteurs: 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
Publié dans: Steroids, 2018, ISSN 0039-128X
Éditeur: Elsevier BV
DOI: 10.1016/j.steroids.2018.02.010

Quantifying fat replacement of muscle by quantitative MRI in muscular dystrophy

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

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

Auteurs: 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
Publié dans: European Journal of Paediatric Neurology, Numéro 21, 2017, Page(s) e238, ISSN 1090-3798
Éditeur: 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

Auteurs: MUDr. Jana Haberlová, Ph.D.
Publié dans: Neurology for Practice, Numéro bimonthly, 2018, Page(s) 2018; 19(2): 108–113, ISSN 1213-1814
Éditeur: NEUROLOGIE PRO PRAXI

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

Auteurs: 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
Publié dans: Neurology, 2019, Page(s) 10.1212/WNL.0000000000008168, ISSN 0028-3878
Éditeur: 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

Auteurs: 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
Publié dans: Pharmacological Research, Numéro 136, 2018, Page(s) 140-150, ISSN 1043-6618
Éditeur: Academic Press
DOI: 10.1016/j.phrs.2018.09.007

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

Auteurs: Olsen, Christina; Head, Ritchie; Knerova, Jane; Larkindale, Jane; Ward, Susan; Spitali, Pietro; Kan, Hermien
Publié dans: Numéro 2, 2021
Éditeur: Zenodo
DOI: 10.5281/zenodo.5668576

RETURNING INDIVIDUAL CLINICAL TRIAL DATA BACK TO PARTICIPANTS

Auteurs: Dr Laurie Conklin; Dr Holly Peay; Gaglianone, Suzanne; Suzie-Ann Bakker; Vroom, Elizabeth; Dr Eric Hoffman; Olsen, Christina
Publié dans: Numéro 3, 2021
Éditeur: Zenodo
DOI: 10.5281/zenodo.4836583

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

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