Award
National Institute of Neurological Disorders and Stroke 1R43NS139721-01A1
New Clinical Biomarkers for GM1 Gamgliosidosis
Recipient
TEGA THERAPEUTICS, INC.
Award Amount
$500,000.00
Ceiling
$500,000.00
Awarded
August 06, 2025
Identifier
1R43NS139721-01A1
NIH funded research project to identify and validate clinical biomarkers for GM1 gangliosidosis to aid in therapy monitoring and newborn diagnosis.
Description
The project aims to identify biomarkers in biological fluids and tissues, and to validate these as tools for monitoring therapy and diagnosing newborns with GM1 gangliosidosis, a lysosomal storage disease characterized by toxic accumulation of gangliosides and glycan metabolites, mainly in the central nervous system. The project involves research including animal studies and enzyme replacement therapy.