LESTER THOMAS W,MOSHASHAEE SAEED,OKHAMAFE AUGUSTUS O,ONEILL CHARLES A
申请号：
IN201717039791
公开号：
IN201717039791A
申请日:
2017.11.08
申请国别(地区):
IN
年份:
2017
代理人:
摘要：
Formulations comprising recombinant human tripeptidyl peptidase 1 (rhTPPl) for intrathecal intracerebroventricular or intraocular administration and kits comprising the same are disclosed. Methods of using rhTPPl 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.
