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

Rezultaty

Develop and publish project website including public summaries of activity

Completion of workshop series

Publikacje

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

Autorzy: 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
Opublikowane w: European Journal of Endocrinology, Numer 2021;184(1), 2021, Strona(/y) 67-79., ISSN 1479-683X
Wydawca: 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

Autorzy: 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
Opublikowane w: Steroids, 2018, ISSN 0039-128X
Wydawca: Elsevier BV
DOI: 10.1016/j.steroids.2018.02.010

Quantifying fat replacement of muscle by quantitative MRI in muscular dystrophy

Autorzy: Jedrzej Burakiewicz, Christopher D. J. Sinclair, Dirk Fischer, Glenn A. Walter, Hermien E. Kan, Kieren G. Hollingsworth
Opublikowane w: Journal of Neurology, Numer 264/10, 2017, Strona(/y) 2053-2067, ISSN 0340-5354
Wydawca: Dr. Dietrich Steinkopff Verlag
DOI: 10.1007/s00415-017-8547-3

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

Autorzy: 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
Opublikowane w: European Journal of Paediatric Neurology, Numer 21, 2017, Strona(/y) e238, ISSN 1090-3798
Wydawca: 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

Autorzy: MUDr. Jana Haberlová, Ph.D.
Opublikowane w: Neurology for Practice, Numer bimonthly, 2018, Strona(/y) 2018; 19(2): 108–113, ISSN 1213-1814
Wydawca: NEUROLOGIE PRO PRAXI

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

Autorzy: 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
Opublikowane w: Neurology, 2019, Strona(/y) 10.1212/WNL.0000000000008168, ISSN 0028-3878
Wydawca: 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

Autorzy: 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
Opublikowane w: Pharmacological Research, Numer 136, 2018, Strona(/y) 140-150, ISSN 1043-6618
Wydawca: 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

Autorzy: Olsen, Christina; Head, Ritchie; Knerova, Jane; Larkindale, Jane; Ward, Susan; Spitali, Pietro; Kan, Hermien
Opublikowane w: Numer 2, 2021
Wydawca: Zenodo
DOI: 10.5281/zenodo.5668576

RETURNING INDIVIDUAL CLINICAL TRIAL DATA BACK TO PARTICIPANTS

Autorzy: Dr Laurie Conklin; Dr Holly Peay; Gaglianone, Suzanne; Suzie-Ann Bakker; Vroom, Elizabeth; Dr Eric Hoffman; Olsen, Christina
Opublikowane w: Numer 3, 2021
Wydawca: Zenodo
DOI: 10.5281/zenodo.4836583

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

Rezultaty

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