Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre,...
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
About this item
Full title
Author / Creator
Mercuri, Eugenio , Vilchez, Juan J , Boespflug-Tanguy, Odile , Zaidman, Craig M , Mah, Jean K , Goemans, Nathalie , Müller-Felber, Wolfgang , Niks, Erik H , Schara-Schmidt, Ulrike , Bertini, Enrico , Comi, Giacomo P , Mathews, Katherine D , Servais, Laurent , Vandenborne, Krista , Johannsen, Jessika , Messina, Sonia , Spinty, Stefan , McAdam, Laura , Selby, Kathryn , Byrne, Barry , Laverty, Chamindra G , Carroll, Kevin , Zardi, Giulia , Cazzaniga, Sara , Coceani, Nicoletta , Bettica, Paolo , McDonald, Craig M , Acsadi, Gyula , Baranello, Giovanni , Blaschek, Astrid , Brandsema, John , Brogna, Claudia , Bruno, Claudio , Connolly, Anne , de Groot, Imelda , De Waele, Liesbeth , Finanger, Erika , Finkel, Richard , Gidaro, Teresa , Guglieri, Michaela , Harper, Amy , Lopez Lobato, Mercedes , Madruga Garrido, Marcos , Magri, Francesca , Manousakis, Georgios , Masson, Riccardo , Monduy, Migvis , Muelas Gomez, Nuria , Munell, Francina , Nascimento, Andres , Nevo, Yoram , Pereon, Yann , Phan, Han , Sansone, Valeria , Scoto, Mariacristina , Vucinic, Dragana , Willis, Tracey and EPIDYS Study Group
Publisher
England: Elsevier Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: Elsevier Ltd
Subjects
More information
Scope and Contents
Contents
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 Duc...
Alternative Titles
Full title
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Authors, Artists and Contributors
Author / Creator
Vilchez, Juan J
Boespflug-Tanguy, Odile
Zaidman, Craig M
Mah, Jean K
Goemans, Nathalie
Müller-Felber, Wolfgang
Niks, Erik H
Schara-Schmidt, Ulrike
Bertini, Enrico
Comi, Giacomo P
Mathews, Katherine D
Servais, Laurent
Vandenborne, Krista
Johannsen, Jessika
Messina, Sonia
Spinty, Stefan
McAdam, Laura
Selby, Kathryn
Byrne, Barry
Laverty, Chamindra G
Carroll, Kevin
Zardi, Giulia
Cazzaniga, Sara
Coceani, Nicoletta
Bettica, Paolo
McDonald, Craig M
Acsadi, Gyula
Baranello, Giovanni
Blaschek, Astrid
Brandsema, John
Brogna, Claudia
Bruno, Claudio
Connolly, Anne
de Groot, Imelda
De Waele, Liesbeth
Finanger, Erika
Finkel, Richard
Gidaro, Teresa
Guglieri, Michaela
Harper, Amy
Lopez Lobato, Mercedes
Madruga Garrido, Marcos
Magri, Francesca
Manousakis, Georgios
Masson, Riccardo
Monduy, Migvis
Muelas Gomez, Nuria
Munell, Francina
Nascimento, Andres
Nevo, Yoram
Pereon, Yann
Phan, Han
Sansone, Valeria
Scoto, Mariacristina
Vucinic, Dragana
Willis, Tracey
EPIDYS Study Group
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_2973103924
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2973103924
Other Identifiers
ISSN
1474-4422,1474-4465
E-ISSN
1474-4465
DOI
10.1016/S1474-4422(24)00036-X
