Lysosomal Disease Treatments Market: Introduction
Lysosomal storage disorders cause several diseases. Lysosomal storage diseases (LSDs) are a large group of disorders caused by a deficiency of specific enzymes responsible for the degradation of substances present in lysosomes. They affect the lysosome — a structure in cells that breaks down substances, such as proteins, carbohydrates, and old cell parts so the body can recycle them. The purpose of substrate reduction therapy is to down regulate the formation of the lysosomal substance to a rate at which the residual enzyme activity can catabolize the stored and de novo produced lysosomal substrate. Chemical chaperone therapy is based on small molecules able to bind and stabilize the misfolded enzymes.
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Clinical signs characterizing different diseases depend on the quantity and type of accumulated substance; in general, the disease is named after the type of undegraded substrate. Most LSDs have an autosomal recessive inheritance pattern. Mucopolysaccharidosis type II, Fabry disease, and Danon disease are X-linked. Different therapeutic approaches were used in the past to address the underlying causes of the diseases: infusion of plasma or plasma fractions, intravenous injection of exogenous enzymes extracted from human tissues, infusion of leukocytes and implantation of skin fibroblasts or amniotic cells. People with these disorders are missing important enzymes (proteins that speed up reactions in the body). Without the enzymes, the lysosome is not able to break down these substances.
Key Drivers of Global Lysosomal Disease Treatments Market
Demand for orphan drug status
Current economic situation, along with growing generic competition has shifted the focus of pharmaceutical companies from essential medicines to a new business model – niche busters, also called orphan drugs
Orphan drugs may help pharma companies to reduce the impact of revenue loss caused by patent expiries of blockbuster drugs
Rare diseases in small patient population, thus “orphaned” by the pharmaceutical industry having but a few approved drug treatment options available are called orphan diseases
Orphan diseases are often so rare that a physician may observe only one case a year or less. Academic physician-scientists have tried to fill this therapy vacuum by working on developing orphan drugs.
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Key Players Operating in Global Market
The global lysosomal disease treatments market is highly concentrated due to the presence of a few key players. Several manufacturers hold major share of the market in their respective regions. Leading players operating in the global lysosomal disease treatments market are:
- Shire PLC
- Pfizer, Inc.
- Sanofi
- BioMarin Pharmaceutical Inc.
- Actelion Ltd.
- Raptor Pharmaceutical Corp.
- Protalix Biotherapeutics Inc.
- Amicus Therapeutics, Inc.
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