Fellowship · Madison, WI
Sponsored by University of Wisconsin Hospitals and Clinics
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 · 2021 · 399 citations · Jennifer Kwon
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.
Nature medicine · 2022 · 263 citations · Jennifer Kwon
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial.
Nature medicine · 2022 · 236 citations · Jennifer Kwon
An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.
Annals of clinical and translational neurology · 2022 · 31 citations · Jennifer Kwon
Newborn Screening for Spinal Muscular Atrophy: Variations in Practice and Early Management of Infants with Spinal Muscular Atrophy in the United States.
International journal of neonatal screening · 2024 · 18 citations · Cameron Crockett
Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy.
Muscle & nerve · 2021 · 47 citations ·
Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Nature medicine · 2026 · 10 citations · Jennifer Kwon
Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series.
BMC neurology · 2025 · 9 citations · Jennifer Kwon