Sitra Tauscher‐Wisniewski

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

Richard S. Finkel, Claudia A. Chiriboga, Anne M. Connolly et al. · The Lancet Neurology · 2021 · 480 citations

Medicine, Genetics, Molecular Genetics +14

Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy

Deepa H. Chand, F. Mohr, Hugh J. McMillan et al. · Journal of Hepatology · 2020 · 297 citations · Full text

Medicine, Pharmacology, Toxicology +17

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

Kevin A. Strauss, Michelle A. Farrar, Francesco Muntoni et al. · Nature Medicine · 2022 · 295 citations · Full text

Medicine, Genetics, Pediatrics +13

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

Kevin A. Strauss, Michelle A. Farrar, Francesco Muntoni et al. · Nature Medicine · 2022 · 276 citations · Full text

Medicine, Genetics, Pediatrics +16

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 · 256 citations

Medicine, Neurology, Neuroscience +12