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European Archives of Oto-Rhino-Laryngology : Official Journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : Affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery|September 4, 2024
Fiberoptic endoscopic evaluation of swallowing (FEES) in children with spinal muscular atrophy type 1: feasibility, swallowing safety and efficacy, and dysphagia phenotypeFrancesco Mozzanica, Nicole Pizzorni, Marco Gitto, et al.
Clinical Epigenetics|December 4, 2025
Histone deacetylases in Duchenne muscular dystrophy: a role in the mechanism of disease and a target for inhibitionMariarita Bertoldi, Emilio Albamonte, Luca Bello, et al.
Muscle & Nerve|February 4, 2020
Evaluation of body composition as a potential biomarker in spinal muscular atrophyGiovanni Baranello, Ramona De Amicis, Maria Teresa Arnoldi, et al.
Neuromuscular Disorders : NMD|May 9, 2026
Swallowing assessment in spinal muscular atrophy type 1: a real-world study of dysphagia in children receiving disease-modifying therapiesSofia Gandolfi, Claudia Dosi, Maria Teresa Arnoldi, et al.
The Journal of Clinical Investigation|September 16, 2025
Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophyCrystal M Proud, Richard S Finkel, Julie A Parsons, et al.
Clinical Nutrition (Edinburgh, Scotland)|March 21, 2021
Predictive fat mass equations for spinal muscular atrophy type I children: Development and internal validationAndrea Foppiani, Ramona De Amicis, Alessandro Leone, et al.
Biomedicines|January 30, 2020
Circulating MyomiRs as Potential Biomarkers to Monitor Response to Nusinersen in Pediatric SMA PatientsSilvia Bonanno, Stefania Marcuzzo, Claudia Malacarne, et al.
Orphanet Journal of Rare Diseases|July 29, 2022
Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter studyClaude Cances, Dmitry Vlodavets, Giacomo Pietro Comi, et al.
The Lancet. Neurology|September 18, 2021
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trialEugenio Mercuri, Francesco Muntoni, Giovanni Baranello, et al.
Frontiers in Cellular Neuroscience|August 29, 2022
Identification of a cytokine profile in serum and cerebrospinal fluid of pediatric and adult spinal muscular atrophy patients and its modulation upon nusinersen treatmentSilvia Bonanno, Paola Cavalcante, Erika Salvi, et al.
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