cerliponase alfa

Cerliponase alfa is a specialized enzyme replacement therapy designed to treat a rare genetic disorder called CLN2 disease, which causes progressive brain deterioration in children. In this condition, a defective gene leads to a deficiency of a natural enzyme called tripeptidyl peptidase 1 (TPP1), resulting in the buildup of harmful substances in brain cells. Cerliponase alfa replaces the missing enzyme, helping to slow the disease's progression. It is administered directly into the cerebrospinal fluid via a surgically implanted device, aiming to protect brain function and improve quality of life for affected children.