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The American Journal of Clinical Nutrition|March 8, 2020
Predictive energy equations for spinal muscular atrophy type I childrenSimona Bertoli, Ramona De Amicis, Giorgio Bedogni, et al.
The New England Journal of Medicine|February 24, 2021
Risdiplam in Type 1 Spinal Muscular AtrophyGiovanni Baranello, Basil T Darras, John W Day, et al.
The New England Journal of Medicine|July 28, 2021
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical ControlsBasil T Darras, Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, et al.
Journal of the Neurological Sciences|March 4, 2026
Workshop report: Findings from the 2025 Italian SMAkers Educational Initiative on SMA management in ItalyStefania Corti, Giulio Gadaleta, Ilaria Bitetti, et al.
Annals of Clinical and Translational Neurology|April 17, 2026
Safety and Tolerability of Givinostat: Evidence From Real-World and Clinical PracticeMarika Pane, Anna Capasso, Chiara Arpaia, et al.
European Journal of Neurology|July 15, 2022
Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatmentEugenio Mercuri, Giovanni Baranello, Odile Boespflug-Tanguy, et al.
International Journal of Molecular Sciences|June 2, 2021
Dysregulation of Muscle-Specific MicroRNAs as Common Pathogenic Feature Associated with Muscle Atrophy in ALS, SMA and SBMA: Evidence from Animal Models and Human PatientsClaudia Malacarne, Mariarita Galbiati, Eleonora Giagnorio, et al.
The Lancet. Child & Adolescent Health|July 1, 2026
Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trialMaria Mazurkiewicz-Bełdzińska, Laurent Servais, Giovanni Baranello, et al.
Children (Basel, Switzerland)|May 16, 2023
Upper Limb Changes in DMD Patients Amenable to Skipping Exons 44, 45, 51 and 53: A 24-Month StudyClaudia Brogna, Marika Pane, Giorgia Coratti, et al.
The Lancet. Neurology|October 16, 2022
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trialRiccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose, et al.
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