ANTISENSE OLIGONUCLEOTIDES USEFUL IN TREATMENT OF POMPE DISEASE
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.
申请公布号
WO2015190922(A1)
申请公布日期
2015.12.17
申请号
WO2015NL50421
申请日期
2015.06.10
申请人
ERASMUS UNIVERSITY MEDICAL CENTER ROTTERDAM
发明人
BERGSMA, ATZE JACOBUS;VAN DER WAL, ERIK;PIJNAPPEL, WILHELMUS WENCESLAUS MATTHIAS;VAN DER PLOEG, ANTJE TJITSKE;REUSER, ARNOLDUS
