Silvana De Lucia

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

Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy

Eve Fouarge, Arnaud Monseur, Bruno Boulanger et al. · Orphanet Journal of Rare Diseases · 2021 · 30 citations · Full text

Computing, Biostatistics, Genetics +17

Contribution of mast cells to injury mechanisms in a mouse model of pediatric traumatic brain injury

Raffaella Moretti, Vibol Chhor, Donatella Bettati et al. · Journal of Neuroscience Research · 2016 · 28 citations · Full text

Life Sciences, Immunology, Neuroscience +17

Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping

Eugenio Mercuri, A. Seferian, Laurent Servais et al. · Neuromuscular Disorders · 2023 · 23 citations · Full text

Pharmacy, Pediatrics, Pharmacokinetics +9

Ketogenic diet therapies in France: State of the use in 2018

Blandine Dozières‐Puyravel, Laurent François, Silvana De Lucia et al. · Epilepsy & Behavior · 2018 · 15 citations

Agriculture, Nutrition, Nutritional Sciences +9