Bergsma, Atze Jacobus,Van Der Wal, Erik,Pijnappel, Wilhelmus Wenceslaus Matthias,Van Der Ploeg, Antje Tjitske,Reuser, Arnoldus
申请号：
AU2015272128
公开号：
AU2015272128A1
申请日:
2015.06.10
申请国别(地区):
AU
年份:
2017
代理人:
摘要：
The present invention is directed to antisense oligomeric compounds that may be used in the treatment Pompe disease as well as method for modulating the splicing of the GAA gene and method to treat Pompe disease. Also pharmaceutical compositions comprising the antisense oligomeric compounds are part of the invention.
