Mucopolysaccharidosis Stocks List

Related ETFs - A few ETFs which own one or more of the above listed Mucopolysaccharidosis stocks.

Mucopolysaccharidosis Stocks Recent News

Date Stock Title
Nov 1 RARE What Makes Ultragenyx Pharmaceutical (RARE) an Exciting Investment Opportunity?
Nov 1 BMRN Beyond the Balance Sheet: What SWOT Reveals About Biomarin Pharmaceutical Inc (BMRN)
Oct 31 BMRN BioMarin price target raised to $106 from $104 at UBS
Oct 31 BMRN BioMarin Pharmaceutical Third Quarter 2024 Earnings: Beats Expectations
Oct 30 BMRN BioMarin downgraded at William Blair following narrowing of revenue guidance
Oct 30 BMRN Q3 2024 BioMarin Pharmaceutical Inc Earnings Call
Oct 30 BMRN BioMarin Q3 Earnings & Revenues Beat Estimates, '24 Guidance Raised
Oct 30 BMRN Biomarin Pharmaceutical Inc (BMRN) Q3 2024 Earnings Call Highlights: Record Revenue Growth and ...
Oct 30 BMRN BioMarin Pharmaceutical Inc. (BMRN) Q3 2024 Earnings Call Transcript
Oct 29 BMRN BioMarin (BMRN) Q3 Earnings: Taking a Look at Key Metrics Versus Estimates
Oct 29 BMRN BioMarin Pharmaceutical (BMRN) Tops Q3 Earnings and Revenue Estimates
Oct 29 BMRN BioMarin Pharmaceutical Inc. 2024 Q3 - Results - Earnings Call Presentation
Oct 29 BMRN BioMarin: Q3 Earnings Snapshot
Oct 29 RARE Ultragenyx to Host Conference Call for Third Quarter 2024 Financial Results and Corporate Update
Oct 29 BMRN BioMarin Pharmaceutical Non-GAAP EPS of $0.91 beats by $0.12, revenue of $745.74M beats by $42.43M
Oct 29 BMRN BioMarin Announces 28% Y/Y Total Revenue Growth in the Third Quarter and Increase in Full-year 2024 Guidance; Reaffirms Long-term Guidance and Outlook
Oct 28 BMRN BioMarin Pharmaceutical Q3 2024 Earnings Preview
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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