Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study.
- Voit, Thomas
- Topaloglu, Haluk
- Straub, Volker
- Muntoni, Francesco
- Deconinck, Nicolas
- Campion, Giles
- De Kimpe, Sjef J
- Eagle, Michelle
- Guglieri, Michela
- Hood, Steve
- Liefaard, Lia
- Lourbakos, Afrodite
- Morgan, Allison
- Nakielny, Joanna
- Quarcoo, Naashika
- Ricotti, Valeria
- Rolfe, Katie
- Servais, Laurent
- Wardell, Claire
- Wilson, Rosamund
- Wright, Padraig
- Kraus, John E
DOIAbstract
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.SCOPUS: ar.jinfo:eu-repo/semantics/publishe
Add to ORKG