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About Sanofi Genzyme Glossary of Terms

About Sanofi Genzyme:

As a pioneer and leader in enzyme replacement therapies, Sanofi Genzyme is committed to finding new therapeutic solutions for patients who may have no other alternatives. Our breakthrough research in genetic engineering and recombinant protein manufacturing has enabled us to develop targeted therapies for several previously untreatable LSDs.  More recently we began clinical trials for an investigational therapy for the treatment of nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick Disease Type B. Though it has taken years of commitment from scientists to begin realizing the potential of gene therapy, we believe it will lead to transformational applications for patients in need of treatment options. 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