Simone Morando

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

Eugenio Mercuri, Francesco Muntoni, Giovanni Baranello et al. · The Lancet Neurology · 2021 · 256 citations

Medicine, Neurology, Neuroscience +12

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose et al. · The Lancet Neurology · 2022 · 104 citations · Full text

Medicine, Neurology, Pharmacotherapy +6

Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy

Marika Pane, Giorgia Coratti, Maria Carmela Pera et al. · Annals of Clinical and Translational Neurology · 2022 · 74 citations · Full text

Medicine, Neurology, Rehabilitation Medicine +17

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

Claudia A. Chiriboga, Claudio Bruno, Tina Duong et al. · Neurology and Therapy · 2023 · 54 citations · Full text

Medicine, Neurology, Pharmacology +16

Nusinersen in pediatric and adult patients with type III spinal muscular atrophy

Maria Carmela Pera, Giorgia Coratti, Francesca Bovis et al. · Annals of Clinical and Translational Neurology · 2021 · 49 citations · Full text

Medicine, Neurology, Pediatrics +14