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Last Updated: 10/31/2025

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Found 659 publications

AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial.

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

Neuroimaging spectrum of GM1 gangliosidosis with description of novel imaging signs.

Neuroimaging spectrum of GM1 gangliosidosis with description of novel imaging signs.

Journal: AJNR. American journal of neuroradiology
Published: June 09, 2025

Brain Age Prediction in Type II GM1 Gangliosidosis.

Brain Age Prediction in Type II GM1 Gangliosidosis.

Journal: medRxiv : the preprint server for health sciences
Published: May 02, 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].

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

Corrigendum: Infection of a β-galactosidase-deficient mouse strain with Theiler's murine encephalomyelitis virus reveals limited immunological dysregulations in this lysosomal storage disease.

Corrigendum: Infection of a β-galactosidase-deficient mouse strain with Theiler's murine encephalomyelitis virus reveals limited immunological dysregulations in this lysosomal storage disease.

Journal: Frontiers in immunology
Published: April 11, 2025

Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases.

Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases.

Journal: Molecular genetics and metabolism
Published: March 17, 2025

A Case for Automated Segmentation of MRI Data in Milder Neurodegenerative Diseases.

A Case for Automated Segmentation of MRI Data in Milder Neurodegenerative Diseases.

Journal: medRxiv : the preprint server for health sciences
Published: March 04, 2025

A Case for Automated Segmentation of MRI Data in Neurodegenerative Diseases: Type II GM1 Gangliosidosis.

A Case for Automated Segmentation of MRI Data in Neurodegenerative Diseases: Type II GM1 Gangliosidosis.

Journal: NeuroSci
Published: February 20, 2025

Sinbaglustat ameliorates disease pathology in a murine model of GM1 gangliosidosis without affecting CNS ganglioside levels.

Sinbaglustat ameliorates disease pathology in a murine model of GM1 gangliosidosis without affecting CNS ganglioside levels.

Journal: Neurobiology of disease
Published: February 12, 2025

Editorial: Role of neuroimaging in the diagnosis and treatment of rare diseases.

Editorial: Role of neuroimaging in the diagnosis and treatment of rare diseases.

Journal: Frontiers in neuroimaging
Published: January 24, 2025

Novel insights into pathomechanisms of retinal neuronal degeneration and reactive gliosis in a murine model of GM1-gangliosidosis.

Novel insights into pathomechanisms of retinal neuronal degeneration and reactive gliosis in a murine model of GM1-gangliosidosis.

Journal: Scientific reports
Published: January 10, 2025

Evaluation of the Landscape of Pharmacodynamic Biomarkers in GM1 and GM2 Gangliosidosis.

Evaluation of the Landscape of Pharmacodynamic Biomarkers in GM1 and GM2 Gangliosidosis.

Journal: Clinical and translational science
Published: December 09, 2024
Showing 1-12 of 659

Last Updated: 10/31/2025