Gain - Krabbe Disease
Krabbe disease is a severe neurodegenerative disorder caused by mutations in GALC, the gene that encodes the galactosylceramidase (GALC) enzyme. These mutations cause GALC misfolding and dysfunction, which leads to the toxic buildup up fats in the central nervous system (CNS) and ultimately leads to the demyelination and death of neurons. There is no cure for this disease and most patients will die before the age of two. Gain is developing allosteric regulators to restore GALC function and potentially limit neuronal cell death, potentially providing the first treatment option to patients who are in desperate need.
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