Krabbe Disease, also known as globoid cell leukodystrophy, is a rare and progressive genetic disorder affecting the nervous system. It is caused by mutations in the GALC gene, resulting in the deficiency of an enzyme essential for the breakdown of certain fats. This leads to the accumulation of toxic substances, damaging the protective myelin sheath of nerve cells. Symptoms often appear in infancy and include developmental delays, muscle weakness, vision loss, and seizures. Unfortunately, there is no cure for Krabbe Disease, but treatments such as stem cell transplantation and supportive care can help manage symptoms and improve quality of life for affected individuals. Early diagnosis and intervention are crucial for optimizing outcomes. Visit: https://perspectives.gaintherapeutics.com/leukodystrophy-therapy-how-is-gain-therapeutics-developing-first-in-class-treatment-for-krabbe-disease/
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