Thomas Lester,Saeed Moshashaee,Augustus O. Okhamafe,Charles A. O'Neill
申请号：
US16368091
公开号：
US20190216905A1
申请日:
2019.03.28
申请国别(地区):
US
年份:
2019
代理人:
摘要：
Formulations comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) for intrathecal, intracerebroventricular, or intraocular administration, and kits comprising the same, are disclosed. Methods of using rhTPP1 in the prevention and treatment of symptoms of Neuronal Ceroid Lipofuscinosis (CLN2) disease are also disclosed. The formulations and methods are effective in halting the progression of CLN2 disease and may be used to treat subjects having CLN2 or a family history of CLN2.
