Claudia A. Chiriboga

Publications by Year

Research Areas

Neurogenetic and Muscular Disorders Research, RNA modifications and cancer, Congenital Anomalies and Fetal Surgery, RNA Research and Splicing, Prenatal Substance Exposure Effects

Most-Cited Works

• → Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy(2017)2,217 cited
• → Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy(2018)1,401 cited
• → Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study(2016)1,008 cited
• → Results from a phase 1 study of nusinersen (ISIS-SMN _Rx ) in children with spinal muscular atrophy(2016)595 cited
• → 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(2021)456 cited
• → Natural history of infantile‐onset spinal muscular atrophy(2017)401 cited
• → Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial(2022)275 cited
• → Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial(2022)260 cited
• → Nusinersen in later-onset spinal muscular atrophy(2019)258 cited
• → Cerebellar ataxia and coenzyme Q10 deficiency(2003)205 cited