Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study


Voit T., Topaloglu H., Straub V., Muntoni F., Deconinck N., Campion G., ...More

LANCET NEUROLOGY, vol.13, no.10, pp.987-996, 2014 (Peer-Reviewed Journal) identifier

  • Publication Type: Article / Article
  • Volume: 13 Issue: 10
  • Publication Date: 2014
  • Doi Number: 10.1016/s1474-4422(14)70195-4
  • Journal Name: LANCET NEUROLOGY
  • Journal Indexes: Science Citation Index Expanded, Scopus
  • Page Numbers: pp.987-996

Abstract

Background Duchenne muscular dystrophy is caused by dystrophin deficiency and muscle deterioration and preferentially affects boys. Antisense-oligonucleotide-induced exon skipping allows synthesis of partially functional dystrophin. We investigated the efficacy and safety of drisapersen, a 2'-O-methyl-phosphorothioate antisense oligonucleotide, given for 48 weeks.