Tay-Sachs Disease Latest Advances

Intraperitoneal Administration of Adenosine A2A Receptor Antagonist Reduces Neuroinflammatory and Antioxidant Responses in GM2 Gangliosidosis Mouse Model.

Journal: Neuropharmacology

Published: June 18, 2026

Modeling Tay-Sachs Disease in Astrocyte-like Cells Reveals Significant Changes in the Transcriptomic Profile.

Journal: International journal of molecular sciences

Published: May 28, 2026

Late-Onset Tay-Sachs Disease With SMALED-Like Muscle MRI Pattern Despite a Distinct Clinical Phenotype.

Journal: Journal of the peripheral nervous system : JPNS

Published: May 18, 2026

Neurofilament light chain (NfL) as a surrogate outcome measure for GM2 gangliosidoses.

Journal: Journal of neurology

Published: April 27, 2026

Disruptions in speech, language and social communication in lysosomal storage diseases.

Journal: Neuroscience and biobehavioral reviews

Published: April 27, 2026

Extracellular vesicles from inflammatory-primed stromal cells reduce in vitro inflammation in Sandhoff disease model.

Journal: Scientific reports

Published: March 17, 2026

Pyrimethamine-based targeting of HEXA gene mutations in Tay-Sachs disease: a computational analysis.

Journal: Journal of biomolecular structure & dynamics

Published: February 20, 2026

The RAINBOW study: a phase 2 trial evaluating the 3-month pharmacokinetic and pharmacodynamic data and 18-month clinical and safety outcomes of nizubaglustat in GM2 gangliosidoses or Niemann-Pick type C disease.

Journal: Molecular genetics and metabolism

Published: January 30, 2026

Late-onset manifestation of Tay-Sachs disease-A disease of the cerebellum and motor neurons with psychiatric sequelae

Journal: Der Nervenarzt

Published: January 26, 2026

Cerebrospinal delivery of a bidirectional AAV9 vector improves optic nerve and retinal pathology in a sheep model of Tay-Sachs disease.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: January 22, 2026

Correction: Vyas et al. Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis. Int. J. Mol. Sci. 2023, 24, 14611.

Journal: International journal of molecular sciences

Published: January 17, 2026

Bilateral Macular Cherry-Red Spots in Infantile Sandhoff Disease.

Journal: Ophthalmology. Retina

Published: January 15, 2026

Tay-Sachs Disease Latest Advances

Find the Latest Research About Tay-Sachs Disease

Intraperitoneal Administration of Adenosine A2A Receptor Antagonist Reduces Neuroinflammatory and Antioxidant Responses in GM2 Gangliosidosis Mouse Model.

Intraperitoneal Administration of Adenosine A2A Receptor Antagonist Reduces Neuroinflammatory and Antioxidant Responses in GM2 Gangliosidosis Mouse Model.

Modeling Tay-Sachs Disease in Astrocyte-like Cells Reveals Significant Changes in the Transcriptomic Profile.

Modeling Tay-Sachs Disease in Astrocyte-like Cells Reveals Significant Changes in the Transcriptomic Profile.

Late-Onset Tay-Sachs Disease With SMALED-Like Muscle MRI Pattern Despite a Distinct Clinical Phenotype.

Late-Onset Tay-Sachs Disease With SMALED-Like Muscle MRI Pattern Despite a Distinct Clinical Phenotype.

Neurofilament light chain (NfL) as a surrogate outcome measure for GM2 gangliosidoses.

Neurofilament light chain (NfL) as a surrogate outcome measure for GM2 gangliosidoses.

Disruptions in speech, language and social communication in lysosomal storage diseases.

Disruptions in speech, language and social communication in lysosomal storage diseases.

Extracellular vesicles from inflammatory-primed stromal cells reduce in vitro inflammation in Sandhoff disease model.

Extracellular vesicles from inflammatory-primed stromal cells reduce in vitro inflammation in Sandhoff disease model.

Pyrimethamine-based targeting of HEXA gene mutations in Tay-Sachs disease: a computational analysis.

Pyrimethamine-based targeting of HEXA gene mutations in Tay-Sachs disease: a computational analysis.

The RAINBOW study: a phase 2 trial evaluating the 3-month pharmacokinetic and pharmacodynamic data and 18-month clinical and safety outcomes of nizubaglustat in GM2 gangliosidoses or Niemann-Pick type C disease.

The RAINBOW study: a phase 2 trial evaluating the 3-month pharmacokinetic and pharmacodynamic data and 18-month clinical and safety outcomes of nizubaglustat in GM2 gangliosidoses or Niemann-Pick type C disease.

Late-onset manifestation of Tay-Sachs disease-A disease of the cerebellum and motor neurons with psychiatric sequelae

Late-onset manifestation of Tay-Sachs disease-A disease of the cerebellum and motor neurons with psychiatric sequelae

Cerebrospinal delivery of a bidirectional AAV9 vector improves optic nerve and retinal pathology in a sheep model of Tay-Sachs disease.

Cerebrospinal delivery of a bidirectional AAV9 vector improves optic nerve and retinal pathology in a sheep model of Tay-Sachs disease.

Correction: Vyas et al. Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis. Int. J. Mol. Sci. 2023, 24, 14611.

Correction: Vyas et al. Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis. Int. J. Mol. Sci. 2023, 24, 14611.

Bilateral Macular Cherry-Red Spots in Infantile Sandhoff Disease.

Bilateral Macular Cherry-Red Spots in Infantile Sandhoff Disease.