US Pompe Disease Market Key Trends, Size, Growth, Shares And Forecast Research Report 2015-2019
Pompe disease, a type of LSD, is caused by mutations in the GAA gene. These mutations inhibit the mechanism of action of GAA, an enzyme responsible for catabolizing glycogen, resulting in accumulation of glycogen in the lysosomes of the cells. The buildup of glycogen in certain organs and tissues, especially in the muscles, hinders the normal functions of the organs. Pompe disease can be further classified as infantile-onset and adult-onset Pompe disease. As the disease is an autosomal recessive hereditary disorder, both parents are carriers and can pass one copy of the mutated gene to their offspring. The disease affects both men and women.
Market analysts forecast the pompe disease market in the US to grow at a CAGR of 10.68 % over the period 2014-2019.
Covered in this report
This report covers the present scenario and the growth prospects of the pompe disease market in US for the period 2015-2019. To calculate the market size, the report considers revenue from the sales of the drugs available in the US market and the drug candidates that are in various phases of development for the treatment of Pompe disease.
The report also presents the vendor landscape and a corresponding detailed analysis of the major vendors in the market. In addition, it discusses the major drivers that influence the growth and the challenges faced by the vendors and the market at large, as well as the key trends that contribute to the growth the market.
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New report, Pompe Disease Market in the US 2015-2019, has been prepared based on an in-depth market analysis with inputs from industry experts. It also covers the landscape of the market in the US and its growth prospects in the coming years. The report includes a discussion of the key vendors operating in this market.
Key vendors
Amicus Therapeutics
BioMarin Pharmaceutical
Genzyme
Other prominent vendors
Audentes Therapeutics
EpiVax
Oxyrane
Sangamo BioSciences
Valerion Therapeutics
Market driver
Special provision 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
Growing public awareness
For a full, detailed list, view our report
Key questions answered in this report
What will the market size be in 2019 and what will the growth rate be?
What are the key market trends?
What is driving this market?
What are the challenges to market growth?
Who are the key vendors in this market space?
What are the market opportunities and threats faced by the key vendors?
What are the strengths and weaknesses of the key vendors?
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Table Of Content
PART 01: Executive summary
Highlights
PART 02: Scope of the report
Market overview
Top-vendor offerings
PART 03: Market research methodology
Research methodology
Economic indicators
PART 04: Introduction
Key market highlights
PART 05: Market landscape
Market overview
Market size and forecast
Five forces analysis
PART 06: Disease overview
Understanding the disease
Types of Pompe disease
Symptoms
Diagnosis
Etiology and pathogenesis
Epidemiology
Management
Economic burden
PART 07: Orphan drug in the US
Development and regulatory challenges
Orphan drug policies in US
PART 08: Pipeline analysis
Key information on pipeline candidates
PART 09: Market drivers
PART 10: Impact of drivers
PART 11: Market challenges
PART 12: Impact of drivers and challenges
PART 13: Market trends
PART 14: Vendor landscape
Competitive scenario
Market share analysis
Other and future prominent vendors
PART 15: Key vendor analysis
Amicus Therapeutics
BioMarin Pharmaceutical
Genzyme
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