Erik van der Wal,Atze Jacobus Bergsma,Wilhelmus Wenceslaus Matthias Pijnappel,Antje Tjitske van der Ploeg,Arnoldus Reuser
申请号：
US15781809
公开号：
US20180360927A1
申请日:
2015.12.07
申请国别(地区):
US
年份:
2018
代理人:
摘要：
The present invention is direct to the treatment of Pompe disease by administration of an enzyme or nucleic acid encoding for said enzyme suitable for Enzyme Replacement Therapy for Pompe disease in combination with the administration of an antisense oligomeric compound that modulates the splicing of acid alpha-glucosidase (GAA) gene.
