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.
- DNA — deoxyribonucleic acid.
- CNS — central 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.