Global Pompe Disease Treatment Market Research Report Forecast to 2025

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    [title] => Global Pompe Disease Treatment Market Research Report Forecast to 2025
    [upcommingtext] => 
    [seo_url] => global-pompe-disease-treatment-market-research-report-forecast
    [shortdesc] => 
    [meta_title] => Global Pompe Disease Treatment Market Research Report Forecast to 2025
    [keyword] => Global Pompe Disease Treatment Market Research Report Forecast to 2025
    [description] => Pompe Disease Treatment Market Research Report - Global Forecast till 2025
Global Pompe Disease Treatment Market: Information by Type (Late-Onset Pompe Disease, Classic Infantile-Onset Pompe Disease, Non-Classic Infantile-Onset Pompe Disease), by
    [curr] => USD
    [category] => 1200
    [publisher] => 182
    [published_date] => 2019-10-31
    [country] => 230
    [author_name] => 
    [page_num] => 78
    [abstract] => Pompe Disease Treatment Market Research Report - Global Forecast till 2025
Global Pompe Disease Treatment Market: Information by Type (Late-Onset Pompe Disease, Classic Infantile-Onset Pompe Disease, Non-Classic Infantile-Onset Pompe Disease), by Therapy (Enzyme Replacement Therapy (ERT) and Gene Therapy), End User (Hospitals & Clinics and Research & Academic Institutes) and Region (Americas, Europe, Asia-Pacific and the Middle East & Africa) - Forecast till 2025

Market analysis
Pompe disease is a genetic metabolic disorder that is usually seen in infants. This disease is caused due to the mutations in the GAA gene which produces the acid, alpha-glucosidase enzyme, that translates glycogen into a simple form. The absence or mutation in the GAA gene results in the accumulation of glycogen that creates heart problems, muscle weakness, and liver harm that can lead to early death in newborns. Factors such as limited awareness about treatment methods and the high cost of therapy hinder market growth. The key factors that drive the Pompe disease treatment market are increasing government initiatives and increasing R&D by manufacturers. The Global Pompe Disease Treatment Market is likely to register a CAGR of 2.6% to attain USD 1,414.1 Million by the year 2025. The Orphan Products Grants funded 18 new grants from a total of 92 grant applications in 2015, to support around 67 ongoing clinical studies that are related to rare diseases as the government has been taking continuous initiatives for the progress and manufacture of advanced products for the diagnosis and treatment of rare diseases such as Pompe disease. Additionally, the governments of various countries such as Sweden, India, the Netherlands, and South Korea are focusing on approving guidelines for the reimbursement of patients enduring enzyme replacement therapy (ERT) for the treatment of Pompe disease. The State Government of Karnataka along with the Employee State Insurance Corporation provides free of cost ERT to patients with lysosomal storage disorders. Additionally, the Union Ministry of Health and Family Welfare has been taking active initiatives for producing a fund of an amount of 13,940 USD million for the treatment of rare diseases, including lysosomal storage disorders. These growing government initiatives for promoting and advancing Pompe disease treatment solutions are widely expected to spur growth of the global Pompe disease treatment market.

Market segmentation
Global Pompe Disease Treatment Market has been segmented on the different basis. Based on Type the market has been segmented into Late-Onset Pompe Disease, Classic Infantile-Onset Pompe Disease and Non-Classic Infantile-Onset Pompe Disease. Based on Therapy, the Global Pompe Disease Treatment Market has been segmented into Enzyme Replacement Therapy (ERT) and Gene Therapy. Based on End User the market has been segmented into hospitals & Clinics and Research & Academic Institutes. Based on region the market has been segmented into different regions like Americas covering North America ( US and Canada) and South America, Europe covering Germany, UK, France, Spain, Italy and Rest of Europe, Asia-Pacific covering China, India, Japan, Australia & New Zealand, Southeast Asia and Rest of Asia-Pacific, Middle East & Africa. The acid alpha-glucosidase is responsible for breaking glycogen, a complex form of sugar, into its simple form. The absence or mutation in the GAA gene leads to the accumulation of glycogen and damages the cells and Pompe disease is a infrequent genetic condition, caused due to the mutation in the GAA gene, which is responsible for making an enzyme called acid alpha-glucosidase. tissues. There are three chief segmentation of Pompe disease, each differing based on age and severity. These different segments are late-onset Pompe disease, non-classic infantile-onset Pompe disease, and classic infantile-onset Pompe disease. The treatment of Pompe disease is disease-specific and depends on the symptoms of the disease. The global Pompe disease treatment market on the basis of therapy has been divided into, enzyme replacement therapy (ERT), gene therapy, and others. The enzyme replacement therapy (ERT) segment captures the maximum share of the total market as it is the only Food and Drug Administration (FDA) approved therapy for the treatment of Pompe diseases. The Pompe disease treatment market has been fragmented, on the basis of end user, into hospitals & clinics and research & academic institutes. The hospitals & clinics segment was valued at USD 860.8 million in the year 2018, and the research & academic institutes segment accounted for a value of USD 320.7 million in 2018 and are expected to exhibit a CAGR of 2.3% during the forecast period.

Regional analysis
Geographically, the global Pompe disease treatment market, based on region, has been split into different regions like Americas, Europe, Asia-Pacific, and the Middle East & Africa.The Americas is responsible for the major share in the global Pompe disease treatment market owing to the grow in the number of patients with Pompe disease. As per the National Organization for Rare Disorders, the anticipated frequency of the occurrence Pompe disease is approximately 1 in 40,000 in the US. European Pompe disease treatment market is second largest market followed by Asia-Pacific. Europe responsible for the second-major market share in the Pompe disease treatment market owing to factors such as the growing research and development initiatives, support by government for the healthcare sector, and development in settlement policies have improved the market growth. Asia-Pacific is expected to be the fastest-growing region in the global Pompe disease treatment market during the forecast period. increasing healthcare expenditures, growing awareness among patients regarding rare disorders, and improved healthcare are factors which is affecting the the growth of the market. Due to low per capita income and absence of suitable healthcare infrastructure support, the Middle East & Africa responsible for a comparatively lesser market share in the year 2018

Major players
The proposed spectators in global Pompe disease treatment market are hospitals, medical devices companies, Research and academic institutes, software companies Governments, associations, industrial bodies, etc. The major companies functioning in the global Pompe disease treatment market are concentrating on firming their global ways by entering into untouched markets. The projected onlookers of the global Pompe disease treatment market are Market research and consulting service providers, Medical research laboratories and Academic medical institutes and universities, Drug Suppliers, Research and development (R&D) companies, Government research laboratories, Independent research laboratories, Government and independent regulatory authorities. Oxyrane, Valerion Therapeutics, AVROBIO, Inc., and CENTOGENE AG., Amicus Therapeutics, Inc., Genzyme Corporation, Audentes Therapeutics are some of Major players in the global Pompe disease treatment market. The players operating in the global Pompe disease treatment market is focusing on product launches, along with expanding their global footprints by entering untapped markets. [table_of_contents] =>

1 Executive Summary
1.1 Introduction
2 Market Introduction
2.1 Scope of the Study
2.2 Research Objective
2.3 List of Assumptions
3 Research Methodology
3.1 Research Process
3.2 Primary Research
3.3 Secondary Research
3.4 Market Size Estimation
4 Market Dynamics
4.1 Introduction
4.2 Drivers
4.2.1 Increasing Government Initiatives
4.2.2 Rising R&D Activities by Manufacturers
4.3 Restraints
4.3.1 High Cost of Treatment
4.4 Opportunities
4.4.1 Emergence of Immunotherapeutic Approaches
4.5 Challenges
5 Market Factor Analysis
5.1 Value Chain Analysis
5.1.1 R&D and Designing
5.1.2 Manufacturing
5.1.3 Distribution & Sales
5.1.4 Post-Sales Services
5.2 Porters Five Forces Model
5.2.1 Bargaining Power of Suppliers
5.2.2 Bargaining Power of Buyers
5.2.3 Threat of New Entrants
5.2.4 Threat of Substitutes
5.2.5 Intensity of Rivalry
6 Global Pompe Disease Treatment Market, by Type
6.1 Introduction
6.2 Late-Onset Pompe Disease
6.3 Classic Infantile-Onset Pompe Disease
6.4 Non-Classic Infantile-Onset Pompe Disease
7 Global Pompe Disease Treatment Market, by Therapy
7.1 Introduction
7.2 Enzyme Replacement Therapy (ERT)
7.3 Gene Therapy
8 Global Pompe Disease Treatment Market, by End User
8.1 Introduction
8.2 Hospitals & Clinics
8.3 Research & Academic Institutes
9 Global Pompe Disease Treatment Market, by Region
9.1 Introduction
9.2 Americas
9.2.1 North America
9.2.1.1 US
9.2.1.2 Canada
9.2.2 South America
9.3 Europe
9.3.1 Germany
9.3.2 France
9.3.3 UK
9.3.4 Italy
9.3.5 Spain
9.3.6 Rest of Europe
9.4 Asia-Pacific
9.4.1 China
9.4.2 Japan
9.4.3 India
9.4.4 South Korea
9.4.5 Australia
9.4.6 Rest of Asia-Pacific
9.5 Middle East & Africa
9.5.1 Middle East
9.5.2 Africa
10 Competitive Landscape
10.1 Introduction
10.2 Key developments
11 Company Profiles
11.1 Amicus Therapeutics, Inc
11.1.1 Company Overview
11.1.2 Financial Overview
11.1.3 Products/Services Offered
11.1.4 Key Developments
11.1.5 SWOT Analysis
11.1.6 Key Strategies
11.2 Genzyme Corporation
11.2.1 Company Overview
11.2.2 Financial Overview
11.2.3 Products/Services Offered
11.2.4 Key Developments
11.2.5 SWOT Analysis
11.2.6 Key Strategies
11.3 Audentes Therapeutics
11.3.1 Company Overview
11.3.2 Financial Overview
11.3.3 Products/Services Offered
11.3.4 Key Developments
11.3.5 SWOT Analysis
11.3.6 Key Strategies
11.4 Oxyrane
11.4.1 Company Overview
11.4.2 Financial Overview
11.4.3 Products/Services Offered
11.4.4 Key Developments
11.4.5 SWOT Analysis
11.4.6 Key Strategies
11.5 Valerion Therapeutics
11.5.1 Company Overview
11.5.2 Financial Overview
11.5.3 Products/Services Offered (Clinical Study)
11.5.4 Key Developments
11.5.5 SWOT Analysis
11.5.6 Key Strategies
11.6 AVROBIO, Inc.
11.6.1 Company Overview
11.6.2 Financial Overview
11.6.3 Products/Services Offered
11.6.4 Key Developments
11.6.5 SWOT Analysis
11.6.6 Key Strategies
11.7 CENTOGENE AG
11.7.1 Company Overview
11.7.2 Financial Overview
11.7.3 Products/Services Offered
11.7.4 Key Developments
11.7.5 SWOT Analysis
11.7.6 Key Strategies
12 Appendix
12.1 References
12.2 Related Reports [list_of_tables] =>

TABLE 1 LIST OF ASSUMPTIONS
TABLE 2 PRIMARY INTERVIEWS
TABLE 3 GLOBAL POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 4 GLOBAL POMPE DISEASE TREATMENT MARKET, FOR LATE-ONSET POMPE DISEASE, BY REGION 20162025 (USD MILLION)
TABLE 5 GLOBAL POMPE DISEASE TREATMENT MARKET, FOR CLASSIC INFANTILE-ONSET POMPE DISEASE, BY REGION 20162025 (USD MILLION)
TABLE 6 GLOBAL POMPE DISEASE TREATMENT MARKET, FOR NON-CLASSIC INFANTILE-ONSET POMPE DISEASE, BY REGION 20162025 (USD MILLION)
TABLE 7 GLOBAL POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 8 GLOBAL POMPE DISEASE TREATMENT MARKET, FOR ENZYME REPLACEMENT THERAPY (ERT), BY REGION 20162025 (USD MILLION)
TABLE 9 GLOBAL POMPE DISEASE TREATMENT MARKET, FOR GENE THERAPY, BY REGION 20162025 (USD MILLION)
TABLE 10 GLOBAL POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 11 GLOBAL POMPE DISEASE TREATMENT MARKET, FOR HOSPITALS & CLINICS, BY REGION 20162025 (USD MILLION)
TABLE 12 GLOBAL POMPE DISEASE TREATMENT MARKET, FOR RESEARCH & ACADEMIC INSTITUTES, BY REGION 20162025 (USD MILLION)
TABLE 13 GLOBAL POMPE DISEASE TREATMENT MARKET, BY REGION 20162025 (USD MILLION)
TABLE 14 AMERICAS: POMPE DISEASE TREATMENT MARKET, BY REGION 20162025 (USD MILLION)
TABLE 15 AMERICAS: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 16 AMERICAS: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 17 AMERICAS: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 18 NORTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY COUNTRY 20162025 (USD MILLION)
TABLE 19 NORTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 20 NORTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 21 NORTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 22 US: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 23 US: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 24 US: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 25 CANADA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 26 CANADA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 27 CANADA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 28 SOUTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 29 SOUTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 30 SOUTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 31 EUROPE: POMPE DISEASE TREATMENT MARKET, BY COUNTRY/REGION 20162025 (USD MILLION)
TABLE 32 EUROPE: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 33 EUROPE: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 34 EUROPE: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 35 GERMANY: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 36 GERMANY: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 37 GERMANY: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 38 FRANCE: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 39 FRANCE: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 40 FRANCE: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 41 UK: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 42 UK: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 43 UK: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 44 ITALY: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 45 ITALY: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 46 ITALY: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 47 SPAIN: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 48 SPAIN: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 49 SPAIN: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 50 REST OF EUROPE: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 51 REST OF EUROPE: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 52 REST OF EUROPE: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 53 ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY COUNTRY/REGION 20162025 (USD MILLION)
TABLE 54 ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 55 ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 56 ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 57 CHINA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 58 CHINA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 59 CHINA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 60 JAPAN: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 61 JAPAN: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 62 JAPAN: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 63 INDIA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 64 INDIA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 65 INDIA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 66 SOUTH KOREA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 67 SOUTH KOREA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 68 SOUTH KOREA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 69 AUSTRALIA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 70 AUSTRALIA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 71 AUSTRALIA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 72 REST OF ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 73 REST OF ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 74 REST OF ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 75 MIDDLE EAST & AFRICA: POMPE DISEASE TREATMENT MARKET, BY REGION 20162025 (USD MILLION)
TABLE 76 MIDDLE EAST & AFRICA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 77 MIDDLE EAST & AFRICA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 78 MIDDLE EAST & AFRICA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 79 MIDDLE EAST: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 80 MIDDLE EAST: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 81 MIDDLE EAST: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 82 AFRICA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 83 AFRICA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 84 AFRICA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 85 MAJOR PLAYERS IN THE GLOBAL POMPE DISEASE TREATMENT MARKET
TABLE 86 KEY DEVELOPMENTS, BY PLAYERS IN THE MARKET [list_of_figures] =>

FIGURE 1 MARKET SYNOPSIS
FIGURE 2 GLOBAL POMPE DISEASE TREATMENT MARKET: STRUCTURE
FIGURE 3 RESEARCH PROCESS
FIGURE 4 TOP-DOWN AND BOTTOM-UP APPROACHES
FIGURE 5 MARKET DYNAMICS: ANALYSIS OF THE GLOBAL POMPE DISEASE TREATMENT MARKET
FIGURE 6 VALUE CHAIN: GLOBAL POMPE DISEASE TREATMENT MARKET
FIGURE 7 PORTERS FIVE FORCES ANALYSIS: GLOBAL POMPE DISEASE TREATMENT MARKET
FIGURE 8 GLOBAL POMPE DISEASE TREATMENT MARKET, BY TYPE 2018 & 2025 (USD MILLION)
FIGURE 9 GLOBAL POMPE DISEASE TREATMENT MARKET, BY THERAPY 2018 & 2025 (USD MILLION)
FIGURE 10 GLOBAL POMPE DISEASE TREATMENT MARKET, BY END USER 2018 & 2025 (USD MILLION)
FIGURE 11 GLOBAL POMPE DISEASE TREATMENT MARKET SHARE, BY REGION 2018 (%)
FIGURE 12 AMERICAS: POMPE DISEASE TREATMENT MARKET SHARE, BY REGION 2018 (%)
FIGURE 13 NORTH AMERICA: POMPE DISEASE TREATMENT MARKET SHARE, BY COUNTRY 2018 (%)
FIGURE 14 EUROPE: POMPE DISEASE TREATMENT MARKET SHARE, BY COUNTRY/REGION 2018 (%)
FIGURE 15 ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET SHARE, BYCOUNTRY/ REGION 2018 (%)
FIGURE 16 MIDDLE EAST & AFRICA: POMPE DISEASE TREATMENT MARKET SHARE, BY REGION 2018 (%) [sample_page] => [brochure] => [new_price] => PDF-4450-ENTERPRISE WIDE LICENCE-6250-ENTERPRISE WIDE LICENCE--MULTI USER LICENCE--ENTERPRISE WIDE LICENCE--ENTERPRISE WIDE LICENCE--Multi User Licence- )
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Pompe Disease Treatment Market Research Report - Global Forecast till 2025
Global Pompe Disease Treatment Market: Information by Type (Late-Onset Pompe Disease, Classic Infantile-Onset Pompe Disease, Non-Classic Infantile-Onset Pompe Disease), by Therapy (Enzyme Replacement Therapy (ERT) and Gene Therapy), End User (Hospitals & Clinics and Research & Academic Institutes) and Region (Americas, Europe, Asia-Pacific and the Middle East & Africa) - Forecast till 2025

Market analysis
Pompe disease is a genetic metabolic disorder that is usually seen in infants. This disease is caused due to the mutations in the GAA gene which produces the acid, alpha-glucosidase enzyme, that translates glycogen into a simple form. The absence or mutation in the GAA gene results in the accumulation of glycogen that creates heart problems, muscle weakness, and liver harm that can lead to early death in newborns. Factors such as limited awareness about treatment methods and the high cost of therapy hinder market growth. The key factors that drive the Pompe disease treatment market are increasing government initiatives and increasing R&D by manufacturers. The Global Pompe Disease Treatment Market is likely to register a CAGR of 2.6% to attain USD 1,414.1 Million by the year 2025. The Orphan Products Grants funded 18 new grants from a total of 92 grant applications in 2015, to support around 67 ongoing clinical studies that are related to rare diseases as the government has been taking continuous initiatives for the progress and manufacture of advanced products for the diagnosis and treatment of rare diseases such as Pompe disease. Additionally, the governments of various countries such as Sweden, India, the Netherlands, and South Korea are focusing on approving guidelines for the reimbursement of patients enduring enzyme replacement therapy (ERT) for the treatment of Pompe disease. The State Government of Karnataka along with the Employee State Insurance Corporation provides free of cost ERT to patients with lysosomal storage disorders. Additionally, the Union Ministry of Health and Family Welfare has been taking active initiatives for producing a fund of an amount of 13,940 USD million for the treatment of rare diseases, including lysosomal storage disorders. These growing government initiatives for promoting and advancing Pompe disease treatment solutions are widely expected to spur growth of the global Pompe disease treatment market.

Market segmentation
Global Pompe Disease Treatment Market has been segmented on the different basis. Based on Type the market has been segmented into Late-Onset Pompe Disease, Classic Infantile-Onset Pompe Disease and Non-Classic Infantile-Onset Pompe Disease. Based on Therapy, the Global Pompe Disease Treatment Market has been segmented into Enzyme Replacement Therapy (ERT) and Gene Therapy. Based on End User the market has been segmented into hospitals & Clinics and Research & Academic Institutes. Based on region the market has been segmented into different regions like Americas covering North America ( US and Canada) and South America, Europe covering Germany, UK, France, Spain, Italy and Rest of Europe, Asia-Pacific covering China, India, Japan, Australia & New Zealand, Southeast Asia and Rest of Asia-Pacific, Middle East & Africa. The acid alpha-glucosidase is responsible for breaking glycogen, a complex form of sugar, into its simple form. The absence or mutation in the GAA gene leads to the accumulation of glycogen and damages the cells and Pompe disease is a infrequent genetic condition, caused due to the mutation in the GAA gene, which is responsible for making an enzyme called acid alpha-glucosidase. tissues. There are three chief segmentation of Pompe disease, each differing based on age and severity. These different segments are late-onset Pompe disease, non-classic infantile-onset Pompe disease, and classic infantile-onset Pompe disease. The treatment of Pompe disease is disease-specific and depends on the symptoms of the disease. The global Pompe disease treatment market on the basis of therapy has been divided into, enzyme replacement therapy (ERT), gene therapy, and others. The enzyme replacement therapy (ERT) segment captures the maximum share of the total market as it is the only Food and Drug Administration (FDA) approved therapy for the treatment of Pompe diseases. The Pompe disease treatment market has been fragmented, on the basis of end user, into hospitals & clinics and research & academic institutes. The hospitals & clinics segment was valued at USD 860.8 million in the year 2018, and the research & academic institutes segment accounted for a value of USD 320.7 million in 2018 and are expected to exhibit a CAGR of 2.3% during the forecast period.

Regional analysis
Geographically, the global Pompe disease treatment market, based on region, has been split into different regions like Americas, Europe, Asia-Pacific, and the Middle East & Africa.The Americas is responsible for the major share in the global Pompe disease treatment market owing to the grow in the number of patients with Pompe disease. As per the National Organization for Rare Disorders, the anticipated frequency of the occurrence Pompe disease is approximately 1 in 40,000 in the US. European Pompe disease treatment market is second largest market followed by Asia-Pacific. Europe responsible for the second-major market share in the Pompe disease treatment market owing to factors such as the growing research and development initiatives, support by government for the healthcare sector, and development in settlement policies have improved the market growth. Asia-Pacific is expected to be the fastest-growing region in the global Pompe disease treatment market during the forecast period. increasing healthcare expenditures, growing awareness among patients regarding rare disorders, and improved healthcare are factors which is affecting the the growth of the market. Due to low per capita income and absence of suitable healthcare infrastructure support, the Middle East & Africa responsible for a comparatively lesser market share in the year 2018

Major players
The proposed spectators in global Pompe disease treatment market are hospitals, medical devices companies, Research and academic institutes, software companies Governments, associations, industrial bodies, etc. The major companies functioning in the global Pompe disease treatment market are concentrating on firming their global ways by entering into untouched markets. The projected onlookers of the global Pompe disease treatment market are Market research and consulting service providers, Medical research laboratories and Academic medical institutes and universities, Drug Suppliers, Research and development (R&D) companies, Government research laboratories, Independent research laboratories, Government and independent regulatory authorities. Oxyrane, Valerion Therapeutics, AVROBIO, Inc., and CENTOGENE AG., Amicus Therapeutics, Inc., Genzyme Corporation, Audentes Therapeutics are some of Major players in the global Pompe disease treatment market. The players operating in the global Pompe disease treatment market is focusing on product launches, along with expanding their global footprints by entering untapped markets.


1Executive Summary
1.1Introduction
2Market Introduction
2.1Scope of the Study
2.2Research Objective
2.3List of Assumptions
3Research Methodology
3.1Research Process
3.2Primary Research
3.3Secondary Research
3.4Market Size Estimation
4Market Dynamics
4.1Introduction
4.2Drivers
4.2.1Increasing Government Initiatives
4.2.2Rising R&D Activities by Manufacturers
4.3Restraints
4.3.1High Cost of Treatment
4.4Opportunities
4.4.1Emergence of Immunotherapeutic Approaches
4.5Challenges
5Market Factor Analysis
5.1Value Chain Analysis
5.1.1R&D and Designing
5.1.2Manufacturing
5.1.3Distribution & Sales
5.1.4Post-Sales Services
5.2Porters Five Forces Model
5.2.1Bargaining Power of Suppliers
5.2.2Bargaining Power of Buyers
5.2.3Threat of New Entrants
5.2.4Threat of Substitutes
5.2.5Intensity of Rivalry
6Global Pompe Disease Treatment Market, by Type
6.1Introduction
6.2Late-Onset Pompe Disease
6.3Classic Infantile-Onset Pompe Disease
6.4Non-Classic Infantile-Onset Pompe Disease
7Global Pompe Disease Treatment Market, by Therapy
7.1Introduction
7.2Enzyme Replacement Therapy (ERT)
7.3Gene Therapy
8Global Pompe Disease Treatment Market, by End User
8.1Introduction
8.2Hospitals & Clinics
8.3Research & Academic Institutes
9Global Pompe Disease Treatment Market, by Region
9.1Introduction
9.2Americas
9.2.1North America
9.2.1.1US
9.2.1.2Canada
9.2.2South America
9.3Europe
9.3.1Germany
9.3.2France
9.3.3UK
9.3.4Italy
9.3.5Spain
9.3.6Rest of Europe
9.4Asia-Pacific
9.4.1China
9.4.2Japan
9.4.3India
9.4.4South Korea
9.4.5Australia
9.4.6Rest of Asia-Pacific
9.5Middle East & Africa
9.5.1Middle East
9.5.2Africa
10Competitive Landscape
10.1Introduction
10.2Key developments
11Company Profiles
11.1Amicus Therapeutics, Inc
11.1.1Company Overview
11.1.2Financial Overview
11.1.3Products/Services Offered
11.1.4Key Developments
11.1.5SWOT Analysis
11.1.6Key Strategies
11.2Genzyme Corporation
11.2.1Company Overview
11.2.2Financial Overview
11.2.3Products/Services Offered
11.2.4Key Developments
11.2.5SWOT Analysis
11.2.6Key Strategies
11.3Audentes Therapeutics
11.3.1Company Overview
11.3.2Financial Overview
11.3.3Products/Services Offered
11.3.4Key Developments
11.3.5SWOT Analysis
11.3.6Key Strategies
11.4Oxyrane
11.4.1Company Overview
11.4.2Financial Overview
11.4.3Products/Services Offered
11.4.4Key Developments
11.4.5SWOT Analysis
11.4.6Key Strategies
11.5Valerion Therapeutics
11.5.1Company Overview
11.5.2Financial Overview
11.5.3Products/Services Offered (Clinical Study)
11.5.4Key Developments
11.5.5SWOT Analysis
11.5.6Key Strategies
11.6AVROBIO, Inc.
11.6.1Company Overview
11.6.2Financial Overview
11.6.3Products/Services Offered
11.6.4Key Developments
11.6.5SWOT Analysis
11.6.6Key Strategies
11.7CENTOGENE AG
11.7.1Company Overview
11.7.2Financial Overview
11.7.3Products/Services Offered
11.7.4Key Developments
11.7.5SWOT Analysis
11.7.6Key Strategies
12Appendix
12.1References
12.2Related Reports

List Of Tables



TABLE 1LIST OF ASSUMPTIONS
TABLE 2PRIMARY INTERVIEWS
TABLE 3GLOBAL POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 4GLOBAL POMPE DISEASE TREATMENT MARKET, FOR LATE-ONSET POMPE DISEASE, BY REGION 20162025 (USD MILLION)
TABLE 5GLOBAL POMPE DISEASE TREATMENT MARKET, FOR CLASSIC INFANTILE-ONSET POMPE DISEASE, BY REGION 20162025 (USD MILLION)
TABLE 6GLOBAL POMPE DISEASE TREATMENT MARKET, FOR NON-CLASSIC INFANTILE-ONSET POMPE DISEASE, BY REGION 20162025 (USD MILLION)
TABLE 7GLOBAL POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 8GLOBAL POMPE DISEASE TREATMENT MARKET, FOR ENZYME REPLACEMENT THERAPY (ERT), BY REGION 20162025 (USD MILLION)
TABLE 9GLOBAL POMPE DISEASE TREATMENT MARKET, FOR GENE THERAPY, BY REGION 20162025 (USD MILLION)
TABLE 10GLOBAL POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 11GLOBAL POMPE DISEASE TREATMENT MARKET, FOR HOSPITALS & CLINICS, BY REGION 20162025 (USD MILLION)
TABLE 12GLOBAL POMPE DISEASE TREATMENT MARKET, FOR RESEARCH & ACADEMIC INSTITUTES, BY REGION 20162025 (USD MILLION)
TABLE 13GLOBAL POMPE DISEASE TREATMENT MARKET, BY REGION 20162025 (USD MILLION)
TABLE 14AMERICAS: POMPE DISEASE TREATMENT MARKET, BY REGION 20162025 (USD MILLION)
TABLE 15AMERICAS: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 16AMERICAS: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 17AMERICAS: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 18NORTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY COUNTRY 20162025 (USD MILLION)
TABLE 19NORTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 20NORTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 21NORTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 22US: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 23US: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 24US: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 25CANADA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 26CANADA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 27CANADA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 28SOUTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 29SOUTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 30SOUTH AMERICA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 31EUROPE: POMPE DISEASE TREATMENT MARKET, BY COUNTRY/REGION 20162025 (USD MILLION)
TABLE 32EUROPE: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 33EUROPE: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 34EUROPE: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 35GERMANY: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 36GERMANY: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 37GERMANY: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 38FRANCE: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 39FRANCE: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 40FRANCE: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 41UK: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 42UK: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 43UK: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 44ITALY: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 45ITALY: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 46ITALY: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 47SPAIN: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 48SPAIN: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 49SPAIN: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 50REST OF EUROPE: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 51REST OF EUROPE: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 52REST OF EUROPE: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 53ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY COUNTRY/REGION 20162025 (USD MILLION)
TABLE 54ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 55ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 56ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 57CHINA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 58CHINA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 59CHINA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 60JAPAN: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 61JAPAN: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 62JAPAN: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 63INDIA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 64INDIA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 65INDIA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 66SOUTH KOREA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 67SOUTH KOREA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 68SOUTH KOREA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 69AUSTRALIA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 70AUSTRALIA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 71AUSTRALIA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 72REST OF ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 73REST OF ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 74REST OF ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 75MIDDLE EAST & AFRICA: POMPE DISEASE TREATMENT MARKET, BY REGION 20162025 (USD MILLION)
TABLE 76MIDDLE EAST & AFRICA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 77MIDDLE EAST & AFRICA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 78MIDDLE EAST & AFRICA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 79MIDDLE EAST: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 80MIDDLE EAST: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 81MIDDLE EAST: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 82AFRICA: POMPE DISEASE TREATMENT MARKET, BY TYPE 20162025 (USD MILLION)
TABLE 83AFRICA: POMPE DISEASE TREATMENT MARKET, BY THERAPY 20162025 (USD MILLION)
TABLE 84AFRICA: POMPE DISEASE TREATMENT MARKET, BY END USER 20162025 (USD MILLION)
TABLE 85MAJOR PLAYERS IN THE GLOBAL POMPE DISEASE TREATMENT MARKET
TABLE 86KEY DEVELOPMENTS, BY PLAYERS IN THE MARKET

List Of Figures



FIGURE 1MARKET SYNOPSIS
FIGURE 2GLOBAL POMPE DISEASE TREATMENT MARKET: STRUCTURE
FIGURE 3RESEARCH PROCESS
FIGURE 4TOP-DOWN AND BOTTOM-UP APPROACHES
FIGURE 5MARKET DYNAMICS: ANALYSIS OF THE GLOBAL POMPE DISEASE TREATMENT MARKET
FIGURE 6VALUE CHAIN: GLOBAL POMPE DISEASE TREATMENT MARKET
FIGURE 7PORTERS FIVE FORCES ANALYSIS: GLOBAL POMPE DISEASE TREATMENT MARKET
FIGURE 8GLOBAL POMPE DISEASE TREATMENT MARKET, BY TYPE 2018 & 2025 (USD MILLION)
FIGURE 9GLOBAL POMPE DISEASE TREATMENT MARKET, BY THERAPY 2018 & 2025 (USD MILLION)
FIGURE 10GLOBAL POMPE DISEASE TREATMENT MARKET, BY END USER 2018 & 2025 (USD MILLION)
FIGURE 11GLOBAL POMPE DISEASE TREATMENT MARKET SHARE, BY REGION 2018 (%)
FIGURE 12AMERICAS: POMPE DISEASE TREATMENT MARKET SHARE, BY REGION 2018 (%)
FIGURE 13NORTH AMERICA: POMPE DISEASE TREATMENT MARKET SHARE, BY COUNTRY 2018 (%)
FIGURE 14EUROPE: POMPE DISEASE TREATMENT MARKET SHARE, BY COUNTRY/REGION 2018 (%)
FIGURE 15ASIA-PACIFIC: POMPE DISEASE TREATMENT MARKET SHARE, BYCOUNTRY/ REGION 2018 (%)
FIGURE 16MIDDLE EAST & AFRICA: POMPE DISEASE TREATMENT MARKET SHARE, BY REGION 2018 (%)

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