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

Risultati finali

Develop and publish project website including public summaries of activity

Completion of workshop series

Pubblicazioni

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

Autori: 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
Pubblicato in: European Journal of Endocrinology, Numero 2021;184(1), 2021, Pagina/e 67-79., ISSN 1479-683X
Editore: 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

Autori: 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
Pubblicato in: Steroids, 2018, ISSN 0039-128X
Editore: Elsevier BV
DOI: 10.1016/j.steroids.2018.02.010

Quantifying fat replacement of muscle by quantitative MRI in muscular dystrophy

Autori: Jedrzej Burakiewicz, Christopher D. J. Sinclair, Dirk Fischer, Glenn A. Walter, Hermien E. Kan, Kieren G. Hollingsworth
Pubblicato in: Journal of Neurology, Numero 264/10, 2017, Pagina/e 2053-2067, ISSN 0340-5354
Editore: Dr. Dietrich Steinkopff Verlag
DOI: 10.1007/s00415-017-8547-3

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

Autori: 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
Pubblicato in: European Journal of Paediatric Neurology, Numero 21, 2017, Pagina/e e238, ISSN 1090-3798
Editore: 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

Autori: MUDr. Jana Haberlová, Ph.D.
Pubblicato in: Neurology for Practice, Numero bimonthly, 2018, Pagina/e 2018; 19(2): 108–113, ISSN 1213-1814
Editore: NEUROLOGIE PRO PRAXI

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

Autori: 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
Pubblicato in: Neurology, 2019, Pagina/e 10.1212/WNL.0000000000008168, ISSN 0028-3878
Editore: 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

Autori: 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
Pubblicato in: Pharmacological Research, Numero 136, 2018, Pagina/e 140-150, ISSN 1043-6618
Editore: 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

Autori: Olsen, Christina; Head, Ritchie; Knerova, Jane; Larkindale, Jane; Ward, Susan; Spitali, Pietro; Kan, Hermien
Pubblicato in: Numero 2, 2021
Editore: Zenodo
DOI: 10.5281/zenodo.5668576

RETURNING INDIVIDUAL CLINICAL TRIAL DATA BACK TO PARTICIPANTS

Autori: Dr Laurie Conklin; Dr Holly Peay; Gaglianone, Suzanne; Suzie-Ann Bakker; Vroom, Elizabeth; Dr Eric Hoffman; Olsen, Christina
Pubblicato in: Numero 3, 2021
Editore: 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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