Ionis Pharmaceuticals, Inc.;LUDWIG INSTITUTE FOR CANCER RESEARCH
发明人:
申请号:
EP17783110.4
公开号:
EP3443094A4
申请日:
2017.04.13
申请国别(地区):
EP
年份:
2019
代理人:
摘要:
Provided are methods for reducing the amount or activity of C90RF72 RNA, and in certain instances of reducing the amount of C90RF72 protein, in an animal. Such methods are useful to prevent or ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include anxiety, reduced spatial learning, and memory loss. Such neurodegenerative diseases include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).