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

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

LANCET NEUROLOGY, cilt.13, sa.10, ss.987-996, 2014 (SCI-Expanded, Scopus)

Yayın Türü: Makale / Tam Makale
Cilt numarası: 13 Sayı: 10
Basım Tarihi: 2014
Doi Numarası: 10.1016/s1474-4422(14)70195-4
Dergi Adı: LANCET NEUROLOGY
Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus
Sayfa Sayıları: ss.987-996
Hacettepe Üniversitesi Adresli: Evet

Özet

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.