Onasemnogene Abeparvovec-Xioi

Last Updated: 09/26/2026

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Found 169 publications

Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland - part II - expert based recommendations for surveillance and management of side-effects.

Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland - part II - expert based recommendations for surveillance and management of side-effects.

Journal: Journal of neuromuscular diseases

Published: August 27, 2026

289th ENMC international workshop: assessing and managing emerging AAV related toxicities after gene therapy for neuromuscular disorders, 26 - 28 September 2025, Hoofddorp, The Netherlands.

289th ENMC international workshop: assessing and managing emerging AAV related toxicities after gene therapy for neuromuscular disorders, 26 - 28 September 2025, Hoofddorp, The Netherlands.

Journal: Neuromuscular disorders : NMD

Published: July 10, 2026

External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalities.

External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalities.

Journal: Molecular therapy. Advances

Published: May 29, 2026

Trends in the Engineering of Adeno-Associated Virus (AAV) for Precision Gene Delivery to the Central Nervous System (CNS).

Trends in the Engineering of Adeno-Associated Virus (AAV) for Precision Gene Delivery to the Central Nervous System (CNS).

Journal: International journal of molecular sciences

Published: May 07, 2026

Adeno-Associated Virus Gene Delivery to Hard-to-Transduce Tissues: Biological Barriers, Engineering Strategies, and Clinical Challenges.

Adeno-Associated Virus Gene Delivery to Hard-to-Transduce Tissues: Biological Barriers, Engineering Strategies, and Clinical Challenges.

Journal: Human gene therapy

Published: April 04, 2026

CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges.

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

Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series.

Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series.

Journal: Advances in therapy

Published: December 12, 2025

Vulnerability, protection and fairness: ethical and regulatory limits in small-N gene therapy trials.

Vulnerability, protection and fairness: ethical and regulatory limits in small-N gene therapy trials.

Journal: Trials

Published: December 11, 2025

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan.

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan.

Journal: Annals of clinical and translational neurology

Published: December 04, 2025

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.

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

Modeling the Budget Impact of Disease-Modifying Therapies in Infantile-Onset Spinal Muscular Atrophy.

Modeling the Budget Impact of Disease-Modifying Therapies in Infantile-Onset Spinal Muscular Atrophy.

Journal: Clinical drug investigation

Published: November 21, 2025

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

Showing 1-12 of 169

Last Updated: 09/26/2026