Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial
The Lancet Neurology
Fecha de publicación: 1 de abril de 2020
Autores: Prof John W Day, MD , Prof Richard S Finkel, MD, Prof Claudia A Chiriboga, MD, Prof Anne M Connolly, MD,Prof Thomas O Crawford, MD,Prof Basil T Darras, MD,Prof Susan T Iannaccone, MD, Prof Nancy L Kuntz, MD, Loren D M Peña, MD, Perry B Shieh, MD, Edward C Smith, MD, Prof Jennifer M Kwon, MD,Craig M Zaidman, MD,Meredith Schultz, MD, Douglas E Feltner, MD, Sitra Tauscher-Wisniewski, MD, Haojun Ouyang, PhD,Deepa H Chand, MD,Douglas M Sproule, MD,Thomas A Macek, PhD, Prof Jerry R Mendell, MD
Background: Spinal muscular atrophy type 1 is a motor neuron disorder resulting in death or the need for permanent ventilation by age 2 years. We aimed to evaluate the safety and efficacy of onasemnogene abeparvovec (previously known as AVXS-101), a gene therapy delivering the survival motor neuron gene (SMN), in symptomatic patients (identified through clinical examination) with infantile-onset spinal muscular atrophy.