Mucopolysaccharidosis Stocks List
Symbol | Grade | Name | % Change | |
---|---|---|---|---|
RGNX | D | REGENXBIO Inc. | -2.60 | |
RARE | F | Ultragenyx Pharmaceutical Inc. | 0.71 | |
BMRN | F | BioMarin Pharmaceutical Inc. | -0.44 |
Related Industries: Biotechnology
Symbol | Grade | Name | Weight | |
---|---|---|---|---|
GNOM | F | Global X Genomics & Biotechnology ETF | 8.29 | |
PBE | D | PowerShares Dynamic Biotech & Genome | 3.76 | |
TMFM | A | Motley Fool Mid-Cap Growth ETF | 3.75 | |
GDOC | D | Goldman Sachs Future Health Care Equity ETF | 3.27 | |
FBT | D | First Trust Amex Biotech Index Fund | 3.15 |
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- Mucopolysaccharidosis
Mucopolysaccharidoses are a group of metabolic disorders caused by the absence or malfunctioning of lysosomal enzymes needed to break down molecules called glycosaminoglycans (GAGs). These long chains of sugar carbohydrates occur within the cells that help build bone, cartilage, tendons, corneas, skin and connective tissue. GAGs (formerly called mucopolysaccharides) are also found in the fluids that lubricate joints.
Individuals with mucopolysaccharidosis either do not produce enough of one of the eleven enzymes required to break down these sugar chains into simpler molecules, or they produce enzymes that do not work properly. Over time, these GAGs collect in the cells, blood and connective tissues. The result is permanent, progressive cellular damage which affects appearance, physical abilities, organ and system functioning.
The mucopolysaccharidoses are part of the lysosomal storage disease family, a group of more than 40 genetic disorders that result when the lysosome organelle in animal cells malfunctions. The lysosome can be thought of as the cell's recycling center because it processes unwanted material into other substances that the cell can utilize. Lysosomes break down this unwanted matter via enzymes, highly specialized proteins essential for survival. Lysosomal disorders like mucopolysaccharidosis are triggered when a particular enzyme exists in too small an amount or is missing altogether.
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