Onasemnogene Abeparvovec-Xioi
Last Updated: 02/24/2026
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Found 156 publications
CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges.
Journal: Molecular therapy. Nucleic acids
Published: January 07, 2026
Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidence.
Journal: Journal of neuromuscular diseases
Published: November 24, 2025
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye.
Journal: Human gene therapy
Published: September 26, 2025
Biomarkers of immune dysregulation and posttreatment inflammation in spinal muscular atrophy.
Journal: Proceedings of the National Academy of Sciences of the United States of America
Published: September 23, 2025
Nonclinical toxicity study duration in AAV gene therapy development: Evidence from industry survey supports adequacy of short-term assessments.
Journal: Molecular therapy. Methods & clinical development
Published: August 14, 2025
Reduction in Perioperative Risk in Patients with Spinal Muscular Atrophy Following the Release of Disease-Modifying Therapies: An Analysis of the National Surgical Quality Improvement Program Database.
Journal: Children (Basel, Switzerland)
Published: July 30, 2025
Engineering Targeted Gene Delivery Systems for Primary Hereditary Skeletal Myopathies: Current Strategies and Future Perspectives.
Journal: Biomedicines
Published: June 10, 2025
Gene replacement therapy to restore polyamine metabolism in a Snyder-Robinson syndrome mouse model.
Journal: Methods in enzymology
Published: May 17, 2025
Combination therapies in spinal muscular atrophy: a systematic review.
Journal: European journal of pediatrics
Published: May 02, 2025
Promises past and future - Gene therapy and the actualisation of future expectations.
Journal: Social science & medicine (1982)
Published: April 30, 2025
Real-world outcomes of spinal muscular atrophy treatment with onasemnogene abeparvovec in Croatia: a comprehensive case series and literature review.
Journal: Frontiers in medicine
Published: April 09, 2025
Subacute liver injury in two young infants following gene replacement therapy for spinal muscular atrophy.
Journal: Molecular therapy. Methods & clinical development
Published: February 28, 2025
Last Updated: 02/24/2026