Journal article

Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Abstract:: Background
Duchenne muscular dystrophy, the most common childhood muscular dystrophy, is caused by dystrophin deficiency. Preclinical and phase 2 study data have suggested that givinostat, a histone deacetylase inhibitor, might help to counteract the effects of this deficiency. We aimed to evaluate the safety and efficacy of givinostat in the treatment of Duchenne muscular dystrophy.

Methods
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Publication status:: Published

Peer review status:: Peer reviewed

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APA Style

Mercuri, E., Vilchez, J. J., Boespflug-Tanguy, O., Zaidman, C. M., Mah, J. K., Goemans, N., Müller-Felber, W., Niks, E. H., Schara-Schmidt, U., Bertini, E., Comi, G. P., Mathews, K. D., Servais, L., Vandenborne, K., Johannsen, J., Messina, S., Spinty, S., McAdam, L., Selby, K., … EPIDYS Study Group. (2024). Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurology, 23(4), 393–403.

MLA Style

Mercuri, E, et al. “Safety and Efficacy of Givinostat in Boys with Duchenne Muscular Dystrophy (EPIDYS): a Multicentre, Randomised, Double-Blind, Placebo-Controlled, Phase 3 Trial.” Lancet Neurology, vol. 23, no. 4, 2024, pp. 393–403.

Chicago Style

Mercuri, E, JJ Vilchez, O Boespflug-Tanguy, et al. 2024. “Safety and Efficacy of Givinostat in Boys with Duchenne Muscular Dystrophy (EPIDYS): a Multicentre, Randomised, Double-Blind, Placebo-Controlled, Phase 3 Trial.” Lancet Neurology 23 (4): 393–403.
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Publisher copy:: 10.1016/s1474-4422(24)00036-x

Authors

+ Mercuri, E More by this author

Role:: Author

+ Vilchez, JJ More by this author

Role:: Author

+ Boespflug-Tanguy, O More by this author

Role:: Author

+ Zaidman, CM More by this author

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+ Mah, JK More by this author

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+ Italfarmaco (Italy) More from this funder

Funder identifier:: https://ror.org/05xsppr58

Publisher:: Elsevier
Journal:: Lancet Neurology More from this journal
Volume:: 23
Issue:: 4
Pages:: 393-403
Publication date:: 2024-03-18
Acceptance date:: 2024-01-24
DOI:: 10.1016/s1474-4422(24)00036-x
EISSN:: 1474-4465
ISSN:: 1474-4422
Pmid:: 38508835

Language:: English
Pubs id:: 1987919
Local pid:: pubs:1987919
Deposit date:: 2025-12-11
ARK identifier:: ark:/29072/ora_3cf8b39190a94bc19b7a071570d3eee0

Terms of use

Copyright holder:: Elsevier Ltd
Copyright date:: 2024
Rights statement:: © 2024 Elsevier Ltd. All rights reserved.

Licence:: Terms and Conditions of Use for Oxford University Research Archive

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