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, ss.987-996, 2014 (SCI İndekslerine Giren Dergi) identifier

  • Cilt numarası: 13 Konu: 10
  • Basım Tarihi: 2014
  • Doi Numarası: 10.1016/s1474-4422(14)70195-4
  • Dergi Adı: LANCET NEUROLOGY
  • Sayfa Sayıları: ss.987-996

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.