| • |
Cell Culture
A nutrient-enriched solution that supports the growth of cell lines. |
| • |
Debridement
The process of removing non-living tissue
from burns and other wounds. |
| • |
Enzyme
A protein produced by living cells that causes specific biochemical reactions at body temperatures. |
| • |
Enzyme Cofactor
Naturally occurring molecules that serve to ‘activate’ specific enzymes. |
| • |
Enzyme Replacement Therapy (ERT)
A therapy designed to address the underlying
enzyme deficiency associated with a lysosomal
storage disorder or other disease characterized
by an enzyme deficiency. |
| • |
Eschar
The black crust formed from burned skin tissue. |
| • |
Host Cell
A cell that has been genetically modified
to produce a specific protein and is used
as a ‘seed’ to produce cells which
are identical to it (a cell line). |
| • |
Lysosomal Storage Disorder (LSD)
A disorder characterized by the buildup of substrates within the lysosomes of cells. |
| • |
Lysosome
A cell organelle that digests wastes and materials
within the cell using enzymes. |
| • |
Mucopolysaccharidosis I (MPS I)
A lysosomal storage disorder characterized by a deficiency of the enzyme, alpha-L-iduronidase. |
| • |
Mucopolysaccharidosis VI (MPS VI)
A lysosomal storage disorder characterized by a deficiency of the enzyme, of N-acetylgalactosamine 4-sulfatase (arylsulfatase B). |
| • |
Organelle
A small structure within a cell that performs
a dedicated function, analogous to an organ
in the human body. |
| • |
Orphan Drug Designation
A U.S. Food and Drug Administration designation
conferred upon investigational products that
are being developed for diseases that affect
fewer than 200,000 patients in the United
States. Products with orphan drug status that
are the first to be approved for a specific
indication have seven years of market exclusivity
within the United States. |
| • |
Orphan
Medicinal Product Designation
A European Medical Evaluation Agency designation
conferred upon investigational products for
diseases that affect fewer than five in 10,000
patients in the European Union. Products with
orphan medicinal status that are the first
to be approved for a specific indication have
10 years market exclusivity within the European
Union. |
| • |
Phenylalanine Hydroxylase (PAH)
An enzyme needed to metabolize phenylalanine, an amino acid found in most protein-containing foods. People with the disease PKU lack sufficient quantities of this enzyme. |
