The term lysosomal storage diseases refers to a group of rare inherited
disorders caused by the deficiency of lysosomal enzymes, activator proteins,
proteins required for normal post-translational modification of lysosomal
enzymes or proteins required for intracellular transport between lysosome and
other cellular compartments. These deficiencies result in deficient enzymatic
activity which, in turn, leads to accumulation of partially digested or
undigested macromolecules inside the cell. The clinical manifestations of lysosomal
storage diseases depend on the type of substrate stored, cell types affected by
this storage, and resulting organ involvement. Due to the high variability of
these components, the clinical manifestations of even a single disease vary
from person to person. These symptoms also vary between members of the same
family with identical mutations. The age of onset also varies for lysosomal
storage diseases depending on the rate of substrate accumulation. Lysosomal
storage diseases are classified on the basis of the substrate being accumulated
or on the type of molecular defect associated with the disease. Neuronal
ceroid-lipofuscinoses vary from classical lysosomal storage diseases where the
component that gets accumulated is mitochondrial ATP synthase subunit C or
sphingolipid activator proteins A and D.
Analysts forecast the Global Lysosomal Storage
Diseases market will grow at a CAGR
of 10.01 percent over the period 2013-2018.
The report recognizes the following companies as the key players in the
Global Lysosomal Storage Diseases market: Actelion Pharmaceuticals Ltd.,
BioMarin Pharmaceutical Inc., Genzyme Corp. and Shire plc.
Other Prominent Vendors in the market are: Mylan, Pfizer, Protalix,
Raptor, Recordati, Sigma-Tau
One of the prominent trends in this market is an increase in awareness
among regulatory authorities. Regulatory bodies are steadily working toward
developing guidelines and providing assistance to drug manufacturers to promote
research in this area.
Covered in this
Report
This report covers the present scenario and the growth prospects of the Global Lysosomal Storage Diseases market for the period 2014-2018. To calculate the market size, the report considers revenue from the sales of various drugs available in the market for the management of different types of lysosomal storage diseases, which include:
• Gaucher disease
• Fabry disease
• Mucopolysaccharidosis
• Pompe disease
• Cystinosis
• NPC
This report covers the present scenario and the growth prospects of the Global Lysosomal Storage Diseases market for the period 2014-2018. To calculate the market size, the report considers revenue from the sales of various drugs available in the market for the management of different types of lysosomal storage diseases, which include:
• Gaucher disease
• Fabry disease
• Mucopolysaccharidosis
• Pompe disease
• Cystinosis
• NPC
Global Lysosomal Storage Diseases Market 2014-2018,
has been prepared based on an in-depth market analysis
with inputs from industry experts. The report covers the Americas and the EMEA
and APAC regions; it also covers the Global Lysosomal Storage Diseases market
landscape and its growth prospects in the coming years. The report also
includes a discussion of the key vendors operating in this market.
According to the report, special Provisions for orphan diseases (also
known as rare diseases) such as lysosomal storage diseases is one of the major
drivers. Special incentives in the form of funding, guidance, and market
exclusivity offer an attractive opportunity to vendors for conducting research
in this area.
Further, the report states that one of the main challenges that the
market faces is the limited patient population. The availability of a limited
treatment group may prolong the time period by which a vendor could recover the
Research & Development (R&D) expenses of the drug. Restricted patient
population might also hinder effective evaluation of drugs based on clinical
trials.
