RENNER, WOLFGANG ANDREAS,DUGOVIC, BRANISLAV,BERTOLINI, RETO
申请号:
CA3059321
公开号:
CA3059321A1
申请日:
2018.04.20
申请国别(地区):
CA
年份:
2018
代理人:
摘要:
The present invention relates to a composition comprising an oligomeric compound comprising one or more tricyclo-deoxyribonucleic acid (tc-DNA) nucleosides and one or more lipid moiety, wherein said one or more lipid moiety is covalently linked to said oligomeric compound either directly or via a spacer, and wherein preferably said oligomeric compound comprises from 5 to 40 monomer subunits, as well as pharmaceutical compositions thereof and their uses in the prevention or treatment of neuromuscular or musculoskeletal diseases such as Duchenne muscular dystrophy or Steinert disease.