Last Updated: 09/26/2026
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Found 29 publications
Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management.
Journal: Human gene therapy
Published: February 13, 2026
Author Correction: Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Journal: Nature medicine
Published: January 28, 2026
Onasemnogene abeparvovec gene therapy for treatment of patients with spinal muscular atrophy: Updated real-world practical considerations.
Journal: Journal of neuromuscular diseases
Published: November 10, 2025
Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial.
Journal: Neurology and therapy
Published: September 10, 2025
Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Journal: Nature medicine
Published: July 23, 2025
Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial.
Journal: Nature medicine
Published: July 23, 2025
A prospective, multi-center, observational study of the safety, tolerability and effectiveness of Nusinersen in adult patients with spinal muscular atrophy.
Journal: Neuromuscular disorders : NMD
Published: June 18, 2025
A prospective, multi-center, observational study of the safety, tolerability and effectiveness of Nusinersen in adult patients with spinal muscular atrophy.
Journal: Neuromuscular disorders : NMD
Published: June 18, 2025
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy.
Journal: Neuromuscular disorders : NMD
Published: June 17, 2025
Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy.
Journal: The Journal of clinical investigation
Published: March 28, 2025
Access to novel therapies for Duchenne muscular dystrophy-Insights from expert treating physicians.
Journal: Annals of the Child Neurology Society
Published: May 08, 2024
Last Updated: 09/26/2026