welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!
not yet recruiting

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications

key information

study id #: NCT04179409

condition: Duchenne Muscular Dystrophy

status: not yet recruiting

purpose:

This is an 48-week open-label study to determine the efficacy and safety of casimersen, eteplirsen, or golodirsen for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or 53, respectively. There will be weekly infusions and two muscle biopsies at baseline and at month 12.

intervention: Casimersen, Eteplirsen, Golodirsen

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT04179409

last updated: December 04, 2019