R. Muni Lofra

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

Revised upper limb module for spinal muscular atrophy: 12 month changes

Maria Carmela Pera, Giorgia Coratti, Elena Mazzone et al. · Muscle & Nerve · 2019 · 150 citations · Full text

Medicine, Neurology, Rehabilitation Medicine +15

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

Respiratory Trajectories in Type 2 and 3 Spinal Muscular Atrophy in the iSMAC Cohort Study

Federica Trucco, Deborah Ridout, Mariacristina Scoto et al. · Neurology · 2020 · 80 citations · Full text

Medicine, Pediatrics, Respiratory Therapy +17