Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

The Lancet Neurology

Fecha de publicación:

Autores: Prof Eugenio Mercuri, MD , Prof Francesco Muntoni, MD, Giovanni Baranello, MD, Riccardo Masson, MD, Prof Odile Boespflug-Tanguy, MD, Claudio Bruno, MD, Stefania Corti, MD, Aurore Daron, MD, Prof Nicolas Deconinck, MD Prof Laurent Servais, MD Prof Volker Straub, MD, Haojun Ouyang, PhD, Deepa Chand, MD Sitra Tauscher-Wisniewski, M, Nuno Mendonca, MD, Arseniy Lavrov, MD

DOI: https://doi.org/10.1016/S1474-4422(21)00251-9

Background: Spinal muscular atrophy is a rare, autosomal recessive, neuromuscular disease caused by biallelic loss of the survival motor neuron 1 (SMN1) gene, resulting in motor neuron dysfunction. In this STR1VE-EU study, we aimed to evaluate the safety and efficacy of onasemnogene abeparvovec gene replacement therapy in infants with spinal muscular atrophy type 1, using broader eligibility criteria than those used in STR1VE-US.