Here are definitions of some of the terms used in our mission statement:

  • Rare diseases — those estimated to affect about 200,000 people or fewer out of the population of a country the size of the United States, for example.
  • Viral vector — viruses that have been modified to safely deliver DNA for therapeutic purposes.
  • DNAdeoxyribonucleic acid.
  • CNScentral nervous system, i.e, brain and spinal cord.
  • Various routes — direct infusion into the brain, or the fluid within or surrounding it or the spinal cord.
  • Late pre-clinical — advanced studies in laboratory animals, primarily those required by the FDA before a therapy can be tried in people.
  • FDA – the United States Food and Drug Administration.