Pubertal induction in adolescents with DMD is associated with high satisfaction, gonadotropin release and increased muscle contractile surface area
(si apre in una nuova finestra)
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
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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
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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
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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 childhoodNové možnosti léčby vrozenýchneuromuskulárních 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
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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
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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
