Jena M. Krueger

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

Spinal Muscular Atrophy Update in Best Practices

Mary Schroth, J. Deans, Diana X. Bharucha Goebel et al. · Neurology Clinical Practice · 2024 · 29 citations · Full text

Medicine, Neurology, Neuroscience +10

Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial

Thomas O. Crawford, Eugenio Mercuri, Heike Kölbel et al. · The Lancet Neurology · 2025 · 19 citations · Full text

Medicine, Neurology, Pharmacotherapy +5

Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study

Thomas O. Crawford, Darryl C. De Vivo, Jena M. Krueger et al. · Frontiers in Neurology · 2024 · 17 citations · Full text

Medicine, Pharmacotherapy, Rehabilitation Medicine +17