Alessandra Govoni

Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

Kathrin Meyer, Laura Ferraiuolo, Leah Schmelzer et al. · Molecular Therapy · 2014 · 269 citations · Full text

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 · 237 citations

Gene Therapies, Clinical Trials, Neurology +2

A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

Jerry R. Mendell, Zarife Sahenk, Vinod Malik et al. · Molecular Therapy · 2014 · 229 citations · Full text

Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3

Lorenzo Maggi, Luca Bello, Silvia Bonanno et al. · Journal of Neurology Neurosurgery & Psychiatry · 2020 · 186 citations · Full text

Genotype and phenotype characterization in a large dystrophinopathic cohort with extended follow-up

Francesca Magri, Alessandra Govoni, Maria Grazia D’Angelo et al. · Journal of Neurology · 2011 · 182 citations

Phenotype Characterization, Urology, Genetic Epidemiology +5