Chiara Brusa

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

Feeding difficulties in children and adolescents with spinal muscular atrophy type 2

Renske I. Wadman, Ramona De Amicis, Chiara Brusa et al. · Neuromuscular Disorders · 2020 · 26 citations · Full text

Medicine, Child Health, Pediatrics +12

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

The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy

Francesco Muntoni, Barry J. Byrne, Hugh J. McMillan et al. · Neurology and Therapy · 2024 · 23 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +14

Assessing Cognitive Function in Neuromuscular Diseases: A Pilot Study in a Sample of Children and Adolescents

Rossella D’Alessandro, Neftj Ragusa, Martina Vacchetti et al. · Journal of Clinical Medicine · 2021 · 13 citations · Full text

Medicine, Neurology, Pediatrics +17