Last Updated: 09/26/2026
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Found 22 publications
AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial.
Journal: The New England journal of medicine
Published: February 10, 2026
AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial.
Journal: medRxiv : the preprint server for health sciences
Published: August 06, 2025
Corrigendum to "Natural history progression of MRI brain volumetrics in type II late-infantile and juvenile GM1 gangliosidosis patients" [Molecular Genetics and Metabolism 2025 Mar;144(3):109025].
Journal: Molecular genetics and metabolism
Published: April 17, 2025
A Case for Automated Segmentation of MRI Data in Milder Neurodegenerative Diseases.
Journal: medRxiv : the preprint server for health sciences
Published: March 04, 2025
Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial.
Journal: Nature medicine
Published: December 20, 2024
Natural history progression of MRI brain volumetrics in type II late-infantile and juvenile GM1 gangliosidosis patients.
Journal: Molecular genetics and metabolism
Published: December 01, 2024
Differential Tractography: A Biomarker for Neuronal Function in Neurodegenerative Disease.
Journal: medRxiv : the preprint server for health sciences
Published: October 07, 2024
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases.
Journal: Human gene therapy
Published: September 26, 2024
Differential tractography: an imaging marker for tissue degeneration in neurodegenerative diseases.
Journal: Brain communications
Published: September 11, 2024
Quantitative reliability assessment of brain MRI volumetric measurements in type II GM1 gangliosidosis patients.
Journal: Frontiers in neuroimaging
Published: April 01, 2024
A novel intrasaccular aneurysm device with high complete occlusion rate: initial results in a rabbit model.
Journal: Journal of neurointerventional surgery
Published: May 03, 2023
Last Updated: 09/26/2026