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About Orphazyme Glossary

About Orphazyme:

Orphazyme is a biopharmaceutical company focused on bringing novel treatments to patients living with life-threatening or debilitating rare diseases. Our research focuses on developing therapies for diseases caused by misfolding of proteins and lysosomal dysfunction. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Gaucher disease, sporadic Inclusion Body Myositis (sIBM), and Amyotrophic Lateral Sclerosis (ALS). For more information, please visit Orphazyme.com.


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 Investigational 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