Laura Antonaci

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

Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy

Maria Carmela Pera, Giorgia Coratti, Nicola Forcina et al. · BMC Neurology · 2017 · 221 citations · Full text

Medicine, Neurology, Rehabilitation Medicine +17

Developmental milestones in type I spinal muscular atrophy

Roberto De Sanctis, Giorgia Coratti, Amy Pasternak et al. · Neuromuscular Disorders · 2016 · 136 citations · Full text

Medicine, Neurology, Pediatrics +16

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

Laurent Servais, Eugenio Mercuri, Volker Straub et al. · Nucleic Acid Therapeutics · 2021 · 126 citations · Full text

Medicine, Pharmacotherapy, Rehabilitation Medicine +14

Clinical Variability in Spinal Muscular Atrophy Type <scp>III</scp>

Giorgia Coratti, Sonia Messina, Simona Lucibello et al. · Annals of Neurology · 2020 · 92 citations

Medicine, Biostatistics, Neurology +14