Successes

Successes to date:

  • 4 company members, 12 academic advisors, 1 patient advisor
  • NIH and FDA liaisons assigned and involved
  • 8 datasets in house
  • D-RSC database has been launched on the C-Path Online Data Repository and members have access
  • Duchenne Therapeutic Area User Guide has been developed, the review period has been completed, and publication is targeted for Q3 2017
  • Approval of the Duchenne Muscular Dystrophy (DMD) Charter from the CDISC Therapeutic Area Program Steering Committee (TAPSC) on August 25, 2016
  • Discussions of our planned disease progression model have been published in PLOS Currents Muscular Dystrophy. 2017 Jan 12. Edition 1
  • D-RSC has held 2 annual meetings, with participation from FDA, NIH, disease experts, and consortium members
  • Multiple presentations to various audiences worldwide