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; Eagle, Michelle; Guglieri, Michela; Hood, Steve; Liefaard, Lia; Lourbakos, Afrodite; Morgan, Allison; Ngkielny, Joanna; Quarcoo, Naashika; Ricotti, Valeria; Rolfe, Katie; Servais, Laurent; Wardell, Claire; Wilson, Rosamund; Wright, Padraig; Kraus, John

doi:10.1016/s1474-4422(14)70195-4

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

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

LANCET NEUROLOGY, vol.13, no.10, pp.987-996, 2014 (SCI-Expanded)

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 (SCI-EXPANDED), Scopus
Page Numbers: pp.987-996
Hacettepe University Affiliated: Yes

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.