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Retricted access

Review article

First published 2020-10

Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy

Debra Stevens, Melanie K. Claborn, Brooke L. Gildon [...]

Annals of Pharmacotherapy

Open access

Research article

First published 2021-03-02

Safety Monitoring of Gene Therapy for Spinal Muscular Atrophy with Onasemnogene Abeparvovec –A Single Centre Experience

Johannes Friese, Stephanie Geitmann, Dorothea Holzwarth [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2024-03-05

Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

Laurent Servais, John W. Day, Darryl C. De Vivo [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2023-05-02

Intrathecal Onasemnogene Abeparvovec for Sitting,Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

Richard S. Finkel, Basil T. Darras, Jerry R. Mendell [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2024-2

Onasemnogene Abeparvovec: Post-infusion Efficacy and Safety in Patients With Spinal Muscular Atrophy (SMA)—A Fondazione Policlinico Gemelli IRCCS Experience

Michele Favia, Domenico Tarantino, Lidia Di Cerbo [...]

Hospital Pharmacy

Open access

Research article

First published 2022-07

Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys

Francis Fonyuy Tukov, Keith Mansfield, Mark Milton [...]

Human Gene Therapy

Open access

Research article

First published 2023-07-04

Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment 1

Katlyn E. McGrattan, Richard D. Shell, Rebecca Hurst-Davis [...]

Journal of Neuromuscular Diseases

Retricted access

Review article

First published 2023-02

Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis

Carlos Pascual-Morena, Iván Cavero-Redondo, Maribel Lucerón-Lucas-Torres [...]

Human Gene Therapy

Retricted access

Research article

First published 2020-10

Clinical Course in a Patient With Spinal Muscular Atrophy Type 0 Treated With Nusinersen and Onasemnogene Abeparvovec

Susan E. Matesanz, Candace Curry, Brianna Gross [...]

Journal of Child Neurology

Open access

Research article

First published 2019

AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort

Samiah A. Al-Zaidy, Stephen J. Kolb, Linda Lowes [...]

Journal of Neuromuscular Diseases

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