Vastus Lateralis Fat Fraction is Associated with Functional Efficacy Endpoints in Patients with Duchenne Muscular Dystrophy Treated with Givinostat in EPIDYS
Craig M. McDonald, Francesco Bellanti, Sara Cazzaniga, Paolo Bettica, Ulrike Schara-Schmidt, Eugenio Mercuri, Michela Guglieri, Nuria Muelas, Krista Vandenborne
Givinostat Effect on Respiratory Function in Duchenne Muscular Dystrophy Before and After Ambulation Loss: Results From EPIDYS, LTSE, and PRO-DMD-01 Studies
Craig M. McDonald, Chamindra G. Laverty, Erika L. Finanger, James E. Signorovitch, Andrea Parodi, Federica Alessi, Sara Cazzaniga, Paolo Bettica, Yann Péréon, Eugenio Mercuri
Design of an Observational Study to Assess Real-World Outcomes of Patients with Duchenne Muscular Dystrophy in the US Treated with Givinostat
Erika L. Finanger, Emma Ciafaloni, Tina Duong, Crystal Proud, Carolina Tesi Rocha, Aravindhan Veerapandiyan, Mary Jane Salinas, Sara Cazzaniga, Katiana Gruppioni, Thomas O. Crawford
Long-Term Safety of Givinostat in Patients With Duchenne Muscular Dystrophy: Results From an Open-Label Extension Study
John F. Brandsema, Gyula Acsadi, Nicoletta Coceani, Federica Alessi, Sara Cazzaniga, Paolo Bettica, Erika L. Finanger
Thorough QT Study on the Effect of Therapeutic and Supratherapeutic Dosing of Givinostat in Healthy Volunteers
Eugenio Mercuri, Barry Byrne, Erik H Niks, Ulrike Schara-Schmidt, Tracey Willis, Thierry Le Tourneau, Odile Boespflug-Tanguy, Michela Guglieri, Nuria Muelas Gómez, David Gómez Andrés, Han C. Phan, Sergio César Díaz, John Bourke, Sara Cazzaniga
Cardiac Safety Data for Givinostat in Ambulant Patients with Duchenne Muscular Dystrophy: Results from EPIDYS Study
Eugenio Mercuri, Barry Byrne, Erik H Niks, Ulrike Schara-Schmidt, Tracey Willis, Thierry Le Tourneau, Odile Boespflug-Tanguy, Michela Guglieri, Nuria Muelas Gómez, David Gómez Andrés, Han C. Phan, Sergio César Díaz, John Bourke, Sara Cazzaniga
Efficacy of Givinostat in the Off-Target Population of EPIDYS: A Subgroup Analysis
Richard Finkel, Han C. Phan, Federica Alessi, Sara Cazzaniga, Paolo Bettica, Chamindra G. Laverty
Modeling Disease Progression in Duchenne Muscular Dystrophy: Reduced Decline in Forced Vital Capacity With Givinostat Compared With Standard of Care
Erika L. Finanger, Chamindra G. Laverty, Federica Alessi, Sara Cazzaniga, Paolo Bettica, Anna Largajolli, Francesco Bellanti, Yann Péréon, Craig M. McDonald, Eugenio Mercuri
Patient-Reported Outcomes From a Phase 3 Study of Givinostat in Patients With Duchenne Muscular Dystrophy
Laura McAdam, Federica Alessi, Nuria Muelas, Liesbeth De Waele, Katiana Gruppioni, Sara Cazzaniga, Paolo Bettica, Craig M. Zaidman
Efficacy of Givinostat by Age (6–7 and >7 years): A Post Hoc Analysis of EPIDYS
Chamindra G. Laverty, Federica Alessi, Nicoletta Coceani, Paolo Bettica, Dragana Vucinic