Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trialEugenio Mercuri, Katie Groves, Francesco Muntoni et al.|The Lancet Neurology|2021Cited by 260
Functional changes in Duchenne muscular dystrophyElena Mazzone, Eugenio Mercuri, Gessica Vasco et al.|Neurology|2011Cited by 178
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 TrialLaurent Servais, Frederick J. Schnell, Baoguang Han et al.|Nucleic Acid Therapeutics|2021Cited by 128
A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophyNathalie Goemans, Yasuhiro Takeshima, Eugenio Mercuri et al.|Neuromuscular Disorders|2017Cited by 128
Hammersmith Functional Motor Scale and Motor Function Measure-20 in non ambulant SMA patientsElena Mazzone, Eugenio Mercuri, Rita De Sanctis et al.|Neuromuscular Disorders|2014Cited by 64