Pompe disease, is an autosomal recessive metabolic disorder that damages muscle and nerve cells throughout the body. It is caused by an accumulation of glycogen in the lysosome due to deficiency of the lysosomal acid alpha-glucosidase enzyme. These disease approximately affects one individual in 40,000 in the U.S, however the incidence varies among different ethnic group. Pompe disease can be treated by symptomatic, supportive and disease-specific ways. As the disease involves impairment of cardiac, respiratory and muscular systems, the treatment entails requirement of specialists with expertise in all these disorders. Cardiologists, dieticians, pediatricians, orthopedists and other specialist may need to co-ordinate and design a well collaborated treatment plan for the patient.
Increasing developments in the gene therapy and enzyme replacement therapies are the primary factors driving the growth of Pompe disease Market. However, inflated costs associated with the pompe disease therapy could hinder the market growth opportunities. Moreover, research activities performed to find an effective treatment for the disease and the growth in the special regulatory drug designations for orphan drugs can further create the growth opportunities for pompe disease market
Among the geographies, North America held the largest market share in 2017 and is expected to hold its dominance in pompe disease market over the forecast period. Asia Pacific is expected to grow at the highest CAGR over the forecast period, owing to increasing awareness about the pompe disease & its treatment procedure and rising healthcare infrastructure in Asia Pacific countries.
The report on pompe disease market is a comprehensive study and presentation of drivers, restraints, opportunities, demand factors, market size, forecasts, and trends in the global pompe disease market over the period of 2016 to 2024. Moreover, the report is collective presentation of primary and secondary research findings.
Porter's five forces model in the report provides insights into the competitive rivalry, supplier and buyer positions in the market and opportunities for the new entrants in the global pompe disease market over the period of 2016 to 2024. Further, Growth Matrix given in the report brings an insight on the investment areas that existing or new market players can consider.
Primary research involves extensive interviews and analysis of the opinions provided by the primary respondents.
The primary research starts with identifying and approaching the primary respondents, the primary respondents are approached through:
1. Key Opinion Leaders associated with the author
2. Internal and External subject matter experts
3. Professionals and participants from LinkedIn, Hoovers, Factiva and Bloggers
Primary research respondents typically include:
1. Executives working with leading companies in the market under review
2. Product/brand/marketing managers
3. CXO level executives
4. Regional/zonal/country managers
5. Vice President level executives.
Secondary research involves extensive exploring through the secondary sources of information available in both public domain and paid sources. Each research study is based on over 500 hours of secondary research accompanied by primary research. The information obtained through the secondary sources is validated through the crosscheck on various data sources.
Secondary sources of the data typically include:
1. Company reports and publications
2. Government/institutional publications
3. Trade and associations journals
4. Databases such as WTO, OECD, World Bank, and among others.
5. Websites and publications by research agencies
The global pompe disease market is segmented on the basis of therapy type, molecule type, dosage form and route of administration.
Global Pompe Disease Market by Therapy Type
- Enzyme Replacement Therapy
- Substrate Reduction Therapy
- Small Molecules
- Amicus Therapeutics
- Biomarin Pharmaceuticals
- Audentes Therapeutics
- Valerion Therapeutics
- Sangamo Biosciences
1. Comprehensive analysis of global as well as regional markets of pompe disease market.
2. Complete coverage of all the segments in pompe disease market to analyze the trends, developments in the global market and forecast of market size up to 2024.
3. Comprehensive analysis of the companies operating in global pompe disease market. The company profile includes analysis of product portfolio, revenue, SWOT analysis and latest developments of the company.
4. Growth Matrix presents analysis of the product segments and geographies that market players should focus to invest, consolidate, expand and/or diversify.
1.1. Report Description
1.2. Research Methods
1.3. Research Approaches
2. Executive Summary
3. Global Pompe Disease Market Overview
3.2. Market Dynamics
3.3. Porter's Five Forces Analysis
3.4. Growth Matrix Analysis
3.5. Competitive Landscape in the Global Pompe Disease Market
4. Global Pompe Disease Market by Therapy Type
4.1. Enzyme Replacement Therapy
4.2. Substrate Reduction Therapy
5. Global Pompe Disease Market by Molecule Type
5.2. Small Molecules
6. Global Pompe Disease Market by Dosage Form
7. Global Pompe Disease Market by Route Of Administration
8. Global Pompe Disease Market by Region 2018-2024
8.1. North America
8.1.1. North America Pompe Disease Market by Therapy Type
8.1.2. North America Pompe Disease Market by Molecule Type
8.1.3. North America Pompe Disease Market by Dosage Form
8.1.4. North America Pompe Disease Market by Route Of Administration
8.1.5. North America Pompe Disease Market by Country
8.2.1. Europe Pompe Disease Market by Therapy Type
8.2.2. Europe Pompe Disease Market by Molecule Type
8.2.3. Europe Pompe Disease Market by Dosage Form
8.2.4. Europe Pompe Disease Market by Route Of Administration
8.2.5. Europe Pompe Disease Market by Country
8.3.1. Asia-Pacific Pompe Disease Market by Therapy Type
8.3.2. Asia-Pacific Pompe Disease Market by Molecule Type
8.3.3. Asia-Pacific Pompe Disease Market by Dosage Form
8.3.4. Asia-Pacific Pompe Disease Market by Route Of Administration
8.3.5. Asia-Pacific Pompe Disease Market by Country
8.4.1. RoW Pompe Disease Market by Therapy Type
8.4.2. RoW Pompe Disease Market by Molecule Type
8.4.3. RoW Pompe Disease Market by Dosage Form
8.4.4. RoW Pompe Disease Market by Route Of Administration
8.4.5. RoW Pompe Disease Market by Sub-region
9. Company Covered
9.1. Amicus Therapeutics
9.2. Biomarin Pharmaceuticals
9.4. Audentes Therapeutics
9.7. Valerion Therapeutics
9.8. Sangamo Biosciences