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Pompe Disease Market in the US 2015-2019

  • November 2015
  • -
  • Infiniti Research Limited
  • -
  • 56 pages

About pompe disease
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.

Technavio's 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.

Technavio's 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?

Table Of Contents

Pompe Disease Market in the US 2015-2019
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
PART 16: Appendix
List of abbreviation
PART 17: Explore Technavio

List of Exhibits
Exhibit 01: Product offerings
Exhibit 02: Pompe disease market in US 2014-2019 ($ millions)
Exhibit 03: Drivers and challenges for Pompe disease market in US
Exhibit 04: Five forces analysis
Exhibit 05: Snapshot of Pompe disease
Exhibit 06: Classification of Pompe disease
Exhibit 07: Signs and symptoms of Pompe disease
Exhibit 08: Differential diagnosis for Pompe disease in infants
Exhibit 09: Differential diagnosis for Pompe disease in children and adults
Exhibit 10: Biochemical pathway of Pompe disease
Exhibit 11: Approaches to treat Pompe disease
Exhibit 12: Economic burden: Pompe disease
Exhibit 13: Orphan drug development and regulatory challenges
Exhibit 14: Pipeline portfolio of Pompe disease market in US
Exhibit 15: Impact of drivers
Exhibit 16: Impact of drivers and challenges
Exhibit 17: Region wise sales of Myozyme/Lumizyme 2011-2014 ($ millions)
Exhibit 18: Key takeaways: Genzyme
Exhibit 19: Amicus Therapeutics: Ongoing clinical studies
Exhibit 20: BioMarin: Product segmentation by net revenue 2014
Exhibit 21: BioMarin: Segmentation by revenue 2014
Exhibit 22: BioMarin: Segmentation by revenue 2013 and 2014 ($ millions)
Exhibit 23: BioMarin: Geographical segmentation by net revenue 2014
Exhibit 24: Genzyme: Business segmentation by revenue 2014
Exhibit 25: Genzyme: Business segmentation by revenue 2013 and 2014 ($ billions)

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