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About Orphazyme Glossary

About Orphazyme:

Orphazyme ApS is a Danish biopharmaceutical company, that develops paradigm-changing medicines for the treatment of genetic diseases. The lead program is in development as a treatment for lysosomal storage diseases. This family of genetic disorders consists of more than 45 diseases and includes Niemann-Pick Disease Type C. Lysosomal storage diseases often affect children, most of whom are currently untreatable. For more information, please visit

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


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