welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!
completed

A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

key information

study id #: NCT01254019

condition: Muscular Dystrophies

status: completed

purpose:

The purpose of this study is to determine whether GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.

intervention: GSK2402968 6mg/kg/week

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT01254019

last updated: February 14, 2019

rareRelated