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Last Updated: 10/31/2025
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Found 555 publications
Decoding the Hex-GM2-MGL2 axis in microglia-neuron crosstalk.
Journal: Trends in pharmacological sciences
Published: October 07, 2025
Five-year analysis of efficacy and safety of a bidirectional AAV gene therapy in Tay-Sachs sheep.
Journal: The Journal of clinical investigation
Published: September 30, 2025
Similarities and Differences in the Late-Onset GM2 Gangliosidoses: Tay-Sachs and Sandhoff Diseases.
Journal: medRxiv : the preprint server for health sciences
Published: September 19, 2025
Myotonic Discharges in Infantile Sandhoff Disease.
Journal: The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques
Published: May 21, 2025
Deep Learning Cerebellar Magnetic Resonance Imaging Segmentation in Late-Onset GM2 Gangliosidosis: Implications for Phenotype.
Journal: medRxiv : the preprint server for health sciences
Published: April 29, 2025
Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases.
Journal: Molecular genetics and metabolism
Published: March 17, 2025
A pathogenic alpha synuclein variant exacerbates disease progression in a neuron-specific Gba-KO mouse.
Journal: Neurobiology of disease
Published: February 23, 2025
Late-onset GM2 gangliosidosis: magnetic resonance imaging, diffusion tensor imaging, and correlational fiber tractography differentiate Tay-Sachs and Sandhoff diseases.
Journal: Journal of neurology
Published: January 17, 2025
Tay-Sachs and Sandhoff Diseases: Diffusion tensor imaging and correlational fiber tractography findings differentiate late-onset GM2 Gangliosidosis.
Journal: medRxiv : the preprint server for health sciences
Published: January 13, 2025
Venglustat in GM2 Gangliosidoses and Related Disorders: Results of the AMETHIST Randomized Controlled and Basket Trials.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics
Published: December 23, 2024
Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial.
Journal: Nature medicine
Published: December 20, 2024
Last Updated: 10/31/2025