GM1 Gangliosidosis Latest Advances

Find the Latest Research About GM1 Gangliosidosis

Last Updated: 06/30/2026

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Found 668 publications

Identification of novel compound heterozygous mutations in the GLB1 gene by whole-exome sequencing in a case of infantile GM1 gangliosidosis: a case report.

Identification of novel compound heterozygous mutations in the GLB1 gene by whole-exome sequencing in a case of infantile GM1 gangliosidosis: a case report.

Journal: Frontiers in pediatrics
Published: March 26, 2026

Diagnostic and therapeutic applications of the glycan biomarker H3N2b in GM1 Gangliosidosis.

Diagnostic and therapeutic applications of the glycan biomarker H3N2b in GM1 Gangliosidosis.

Journal: Molecular genetics and metabolism
Published: March 25, 2026

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

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

Journal: The New England journal of medicine
Published: February 10, 2026

Development of Small-Molecule Allosteric Modulators of Beta-Galactosidase (β-Gal) for the Treatment of GM1 Gangliosidosis and Morquio B.

Development of Small-Molecule Allosteric Modulators of Beta-Galactosidase (β-Gal) for the Treatment of GM1 Gangliosidosis and Morquio B.

Journal: International journal of molecular sciences
Published: January 30, 2026

Novel Galactosidase-Beta-1 Variant in Infantile GM1 Gangliosidosis: A Case Report.

Novel Galactosidase-Beta-1 Variant in Infantile GM1 Gangliosidosis: A Case Report.

Journal: Cureus
Published: January 15, 2026

Clinical, Radiological, and Genetic Profiles of Eight Patients with Combined Dystonic Manifestation of Type-III GM1 Gangliosidosis: A Video Case Series from India.

Clinical, Radiological, and Genetic Profiles of Eight Patients with Combined Dystonic Manifestation of Type-III GM1 Gangliosidosis: A Video Case Series from India.

Journal: Tremor and other hyperkinetic movements (New York, N.Y.)
Published: December 08, 2025

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

Alkaline Phosphatase and Infantile GM1 Gangliosidosis: A Simple Biomarker for a Complex Disease?

Alkaline Phosphatase and Infantile GM1 Gangliosidosis: A Simple Biomarker for a Complex Disease?

Journal: JIMD reports
Published: August 01, 2025

From Molecule to Meaning: Neuronopathic Biomarkers and Clinical Relevance in GM1.

From Molecule to Meaning: Neuronopathic Biomarkers and Clinical Relevance in GM1.

Journal: Journal of inherited metabolic disease
Published: July 20, 2025

Lentiviral hematopoietic stem cell gene therapy ameliorates GM1-gangliosidosis in mice.

Lentiviral hematopoietic stem cell gene therapy ameliorates GM1-gangliosidosis in mice.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: July 08, 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
Showing 1-12 of 668

Last Updated: 06/30/2026