Gloria Ferrantini

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

An observational study of functional abilities in infants, children, and adults with type 1 SMA

Marika Pane, Concetta Palermo, Sonia Messina et al. · Neurology · 2018 · 47 citations · Full text

Medicine, Neurology, Pediatrics +13

Assessing Joint Hypermobility in Preschool-Aged Children

Domenico M. Romeo, Simona Lucibello, Elisa Musto et al. · The Journal of Pediatrics · 2016 · 39 citations

Medicine, Child Health, Pediatrics +11

Disorders of early language development in Dravet syndrome

Daniela Chieffo, Domenica Battaglia, Simona Lucibello et al. · Epilepsy & Behavior · 2015 · 23 citations

Education, Linguistics, Pediatrics +14

Ultrasound assessment of diaphragmatic function in type 1 spinal muscular atrophy

Danilo Buonsenso, Beatrice Berti, Concetta Palermo et al. · Pediatric Pulmonology · 2020 · 22 citations

Medicine, Physical Medicine, Neuromusculoskeletal Assessment +14