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Last Updated: 10/31/2025

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Found 151 publications

SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye.

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.

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

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.

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.

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.

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.

Combination therapies in spinal muscular atrophy: a systematic review.

Journal: European journal of pediatrics
Published: May 02, 2025

Real-world outcomes of spinal muscular atrophy treatment with onasemnogene abeparvovec in Croatia: a comprehensive case series and literature review.

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.

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

Costs of Treating Onasemnogene Abeparvovec-Xioi-Induced Liver Injury.

Costs of Treating Onasemnogene Abeparvovec-Xioi-Induced Liver Injury.

Journal: Pharmacology research & perspectives
Published: January 18, 2025

Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series.

Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series.

Journal: BMC neurology
Published: January 17, 2025

Drug-induced autoimmune-like hepatitis: A disproportionality analysis based on the FAERS database.

Drug-induced autoimmune-like hepatitis: A disproportionality analysis based on the FAERS database.

Journal: PloS one
Published: September 05, 2024

Identifying novel response markers for spinal muscular atrophy revealed by targeted proteomics following gene therapy.

Identifying novel response markers for spinal muscular atrophy revealed by targeted proteomics following gene therapy.

Journal: Gene therapy
Published: September 05, 2024
Showing 1-12 of 151

Last Updated: 10/31/2025