Krabbe Disease Treatment: Advances and Challenges in Rare Neurological Care
Krabbe disease, also known as globoid cell leukodystrophy, is a rare, inherited neurodegenerative disorder caused by a deficiency of the enzyme galactocerebrosidase (GALC). This enzyme deficiency leads to the accumulation of toxic substances in the nervous system, resulting in the destruction of myelin—the protective sheath around nerve fibers. Symptoms often manifest in infancy,...
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