The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System
发明人:
申请号：
EP12705552.3
公开号：
EP2672977A1
申请日:
2012.02.08
申请国别(地区):
EP
年份:
2013
代理人:
摘要：
Embodiments of the present invention are directed generally to antisense compounds and compositions for the treatment of muscular dystrophy, and in particular, Duchenne muscular dystrophy (DMD). In one embodiment, the invention is directed to antisense oligonucleotide molecules, pharmaceutical compositions and formulations comprising antisense oligonucleotide molecules, and methods of treating muscular dystrophy related diseases and disorders wherein the antisense oligonucleotide molecules comprises a base sequence selected from the group consisting of SEQ ID NO: 5-8, 10, 12, 14, 16, 24, 27, 28, 34, 35, 37, 40, 42, 44-46, 79, 97, 100, 101, and 116, and combinations thereof.
