Emerging Pharmacological Approaches in Krabbe Disease Treatment: New Hope for Patients
Krabbe disease is a rare, inherited neurodegenerative disorder caused by the deficiency of the enzyme galactocerebrosidase (GALC), leading to the accumulation of toxic substances in the brain and nervous system. This progressive condition results in severe neurological impairment and, ultimately, death. Traditional treatments for Krabbe disease, such as stem cell transplantation, have shown promise, but…