METHODS FOR TREATING CLN2 DISEASE IN PEDIATRIC SUBJECTS

摘要

Provided herein are methods of treating Neuronal Ceroid Lipofuscinosis (CLN2) disease in a subject less than 3 years old. In exemplary embodiments, the method comprises administering to the subject a formulation comprising recombinant human tripeptidyl peptidase-1 (rhTPPl) in an amount effective to treat the CLN2 disease in the subject. Also provided are methods of delaying the onset of CLN2 disease, or a symptom thereof, in a subject less than 3 years old. In exemplary embodiments, the method comprises administering to the subject a formulation comprising recombinant human tripeptidyl peptidase-1 (rhTPPl) in an amount effective to delay the onset of the CLN2 disease or symptom in the subject.