Last Updated: 09/26/2026
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Found 3 publications
Validating a Human Cell Model of Null Galactosylceramidase (GALC) Enzyme Activity That Recapitulates Krabbe Disease.
Journal: Journal of inherited metabolic disease
Published: January 19, 2026
Reduced heparan sulfate levels in cerebrospinal fluid reflect brain neuron correction in Sanfilippo B mice.
Journal: The Journal of clinical investigation
Published: August 28, 2025
Recombinant human alpha-N-acetylglucosamine-6-sulfatase delivered to Sanfilippo D mice with repeated intracerebroventricular injections corrects CNS pathology.
Journal: PloS one
Published: March 03, 2025
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Last Updated: 09/26/2026