Baylor College of Medicine;Ionis Pharmaceuticals, Inc.
发明人:
Susan M. Freier,Huda Y. Zoghbi,Ezequiel Sztainberg
申请号：
US16535888
公开号：
US20200078389A1
申请日:
2019.08.08
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Disclosed herein are compounds and methods for decreasing MECP2 mRNA and protein expression. Such compounds and methods are useful to treat, prevent, or ameliorate MECP2 associated disorders and syndromes. Such MECP2 associated disorders include MECP2 duplication syndrome.
