Glossary of Terms

ASMD – Acid Sphingomyelinase (Niemann-Pick Type A/B & Type B)

EMA – European Medicines Agency (European equivalent of FDA)

ERT – Enzyme Replacement Therapy

FDA – United States Food and Drug Administration

HDAC – Histone Deacetylase Inhibitors

ICV – Intracerebroventricular

IND – Investigational New Drug

i-IND – Individual Investiational New Drug

IRB – Institutional Review Board

NICHD – National Institute of Child Health and Human Development

NIH – National Institutes of Health

NPC – Niemann-Pick disease Type C Disease

TRND – Therapeutics for Rare and Neglected Diseases