Global Pompe Disease Market 2015-2019

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Global Pompe Disease Market 2015-2019
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Description
Report Overview
About Pompe Disease Pompe disease is a type of
LSD. LSD refers to a group of diseases arising
from the accumulation of specific substrates.
LSDs are progressive and have variable age of
onset and clinical symptoms. LSDs are inherited
autosomal recessive disorders. Pompe disease has
two broad sub-types infantile-onset Pompe
disease and adult-onset Pompe disease. Pompe
disease occurs as a result of mutations in the
GAA gene. These mutations are responsible for the
preventing the action of the enzyme acid
alpha-glucosidase, which is responsible for
catabolizing glycogen, thereby allowing sugar to
accumulate and reach toxic levels in the
lysosomes of cells. The buildup of glycogen in
certain organs and tissues, mostly in the
muscles, hinders the normal functions of organs.
Pompe disease is an autosomal recessive
disease.This implies that both the parents pass
on one abnormally mutated copy of the gene to
their child. A parent carrying an abnormal copy
of the gene is known as a carrier. Pompe disease
affects both men and women, irrespective of the
gender. Pompe disease affects people across all
ethnicities and races. Individuals affected with
Pompe disease acquire the disease at birth,
however, the onset of symptoms varies
extensively. More than 300 genetic mutations that
can cause Pompe disease have been identified by
the researchers till date.
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Description
Report Overview
Analysts forecast the Global Pompe Disease market
to grow at a CAGR of 11.79 percent over the
period 2014-2019. Covered in this Report This
report covers the present scenario and the growth
prospects of the Global Pompe Disease market for
the period 2015-2019. To calculate the market
size, the report considers revenue from the sales
of drugs available in the market for the
management of Pompe disease. The report also
presents the vendor landscape and a corresponding
detailed analysis of the major vendors in the
Global Pompe Disease market. The report also
considers the revenue that is likely to be
generated from the drug candidates that are under
various phases of development. In addition, it
discusses the major drivers that influence the
growth of the market, the challenges faced by the
vendors and the market at large, as well as the
key trends that contribute to the growth the
market. Read more details at
http//www.bigmarketresearch.com/global-pompe-dise
ase-2015-2019-market
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Description
Report Overview
Global Pompe Disease Market 2015-2019, 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 Pompe Disease
market landscape and its growth prospects over
the coming years. The report also includes a
discussion of the key vendors operating in this
market. Key Regions Americas APAC EMEA
Key Vendors Amicus Therapeutics BioMarin
Pharmaceutical Genzyme Enquire About Report _at_
http//www.bigmarketresearch.com/report-enquiry/22
1060
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Description
Report Overview
Market Driver Special Provisions for Orphan
Drugs For a full, detailed list, view our
report Market Challenge Limited Patient
Population For a full, detailed list, view our
report Market Trend Regulatory Assistance in
Emerging Nations For a full, detailed list,
view our report
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Table Of Contents
Table Of Content
01.Executive Summary 02. List of
Abbreviations 03. Scope of the Report 04. Market
Research Methodology 05. Introduction 06. Disease
Overview 07. Market Landscape 08. Pipeline
Analysis 09. Geographical Segmentation 10. Buying
Criteria 11. Market Growth Drivers 12. Drivers
and their Impact 13. Market Challenges 14. Impact
of Drivers and Challenges 15. Market Trends 16.
Trends and their Impact 17. Vendor Landscape 18.
Key Vendor Analysis 19. Other Reports in this
Series    
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