Pompe Disease Market by Therapy Type (Enzyme Replacement Therapy and Substrate Reduction Therapy), Molecule Type (Biologics and Small Molecules), Route of Administration (Oral and Parenteral), and Dosage Forms (Solid and Liquid) - Global Opportunity Analysis and Industry Forecast, 2017-2023

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Pompe disease is a disorder caused by the buildup of a complex sugar called glycogen in the body's cells. It is genetically inherited and the accumulation of glycogen in certain organs as well as tissues, especially muscles, impairs their ability to function normally. Pompe diseases affects approximately one individual in 40,000 in the U.S.; however, the incidence varies among different ethnic groups.

Increasing research activities preformed to find an effective treatment for the disease and the growth in the special regulatory drug designations for orphan drugs drive the market. However, inflated costs associated with the Pompe disease therapy restrain the market growth. Moreover, emerging immunotherapeutic approaches for Pompe disease offer lucrative opportunities for the expansion of the market.

The market is segmented on the basis of therapy type, molecule type, route of administration, dosage forms, and region. Based on therapy type, the market is divided into enzyme replacement therapy and substrate reduction therapy. Based on the molecule type, the market is classified into biologics and small molecules. Based on route of administration, the market is bifurcated into oral and parenteral. Based on the dosage forms, the market is categorized into solid and liquid. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

Key players that operate in the market are Amicus Therapeutics, BioMarin Pharmaceutical, Genzyme, Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences, and Valerion Therapeutics.

Key Benefits

The study provides an in-depth analysis of the global Pompe disease market with current trends and future estimations to elucidate imminent investment pockets.
It presents a quantitative analysis from 2016 to 2023 to enable stakeholders to capitalize on prevailing market opportunities.
Key players are profiled and their strategies are thoroughly analyzed, which predicts the competitive outlook of the market.
By region, the market is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

Pompe Disease Market Key Segments:

By Therapy Type

Enzyme Replacement Therapy
Substrate Reduction Therapy

By Molecule Type

Biologics
Small Molecules

By Route of Administration

Oral
Parenteral

By Dosage Forms

Solid
Liquid

By Region

North America
- U.S.
- Canada
- Mexico
Europe
- Germany
- UK
- France
- Spain
- Italy
- Rest of Europe
Asia-Pacific
- Japan
- China
- India
- Australia
- Rest of Asia-Pacific
LAMEA
- Brazil
- Saudi Arabia
- South Africa
- Argentina
- Rest of LAMEA

Chapter: 1 INTRODUCTION

1.1. REPORT DESCRIPTION
1.2. KEY BENEFITS
1.3. KEY MARKET SEGMENTS
1.4. RESEARCH METHODOLOGY

1.4.1. Secondary research
1.4.2. Primary research
1.4.3. Analyst tools & models

Chapter: 2 EXECUTIVE SUMMARY

2.1. CXO PERSPECTIVE

Chapter: 3 MARKET OVERVIEW

3.1. MARKET DEFINITION AND SCOPE
3.2. KEY FINDINGS

3.2.1. Top investment pockets
3.2.2. Top winning strategies

3.3. MARKET SHARE ANALYSIS/TOP PLAYER POSITIONING, 2016
3.4. PORTER’S FIVE FORCES ANALYSIS

3.8. MARKET DYNAMICS
3.8.1. Drivers
3.8.2. Restraints
3.8.3. Opportunities

Chapter: 4 GLOBAL POMPE DISEASE MARKET, BY THERAPY TYPE

4.1. OVERVIEW

4.1.1. Market size and forecast

4.2. ENZYME REPLACEMENT THERAPY

4.2.1. Key market trends
4.2.2. Growth factors and opportunities
4.2.3. Market size and forecast

4.3. SUBSTRATE REDUCTION THERAPY

4.3.1. Key market trends
4.3.2. Growth factors and opportunities
4.3.3. Market size and forecast

Chapter: 5 GLOBAL POMPE DISEASE MARKET, BY MOLECULE TYPE

5.1. OVERVIEW

5.1.1. Market size and forecast

5.2. BIOLOGICS

5.2.1. Key market trends
5.2.2. Growth factors and opportunities
5.2.3. Market size and forecast

5.3. SMALL MOLECULES

5.3.1. Key market trends
5.3.2. Growth factors and opportunities
5.3.3. Market size and forecast

Chapter: 6 GLOBAL POMPE DISEASE MARKET, BY ROUTE OF ADMINISTRATION

6.1. OVERVIEW

6.1.1. Market size and forecast

6.2. ORAL

6.2.1. Key market trends
6.2.2. Growth factors and opportunities
6.2.3. Market size and forecast

6.3. PARENTERAL

6.3.1. Key market trends
6.3.2. Growth factors and opportunities
6.3.3. Market size and forecast

Chapter: 7 GLOBAL POMPE DISEASE MARKET, BY DOSAGE FORMS

7.1. OVERVIEW

7.1.1. Market size and forecast

7.2. SOLID

7.2.1. Key market trends
7.2.2. Growth factors and opportunities
7.2.3. Market size and forecast

7.3. LIQUID

7.3.1. Key market trends
7.3.2. Growth factors and opportunities
7.3.3. Market size and forecast

Chapter: 8 GLOBAL POMPE DISEASE MARKET, BY REGION

8.1. OVERVIEW
8.2. NORTH AMERICA

8.2.1. Key market trends
8.2.2. Key growth factors and opportunities
8.2.3. Market size and forecast

8.2.3.1. U.S. market size and forecast
8.2.3.2. Canada market size and forecast
8.2.3.3. Mexico market size and forecast

8.2.4. North America Pompe Disease Market by Therapy Type
8.2.5. North America Pompe Disease Market by Molecule Type
8.2.6. North America Pompe Disease Market by Route of Administration
8.2.7. North America Pompe Disease Market by Dosage Forms

8.3. EUROPE

8.3.1. Key market trends
8.3.2. Key growth factors and opportunities
8.3.3. Market size and forecast

8.3.3.1. Germany market size and forecast
8.3.3.2. France market size and forecast
8.3.3.3. UK market size and forecast
8.3.3.4. Italy market size and forecast
8.3.3.8. Spain market size and forecast
8.3.3.6. Rest of Europe market size and forecast

8.3.4. Europe Pompe Disease Market by Therapy Type
8.3.5. Europe Pompe Disease Market by Molecule Type
8.3.6. Europe Pompe Disease Market by Route of Administration
8.3.7. Europe Pompe Disease Market by Dosage Forms

8.4. ASIA-PACIFIC

8.4.1. Key market trends
8.4.2. Key growth factors and opportunities
8.4.3. Market size and forecast

8.4.3.1. Japan market size and forecast
8.4.3.2. China market size and forecast
8.4.3.3. Australia market size and forecast
8.4.3.4. India market size and forecast
8.4.3.8. Rest of Asia-Pacific market size and forecast

8.4.4. Asia-Pacific Pompe Disease Market by Therapy Type
8.4.5. Asia-Pacific Pompe Disease Market by Molecule Type
8.4.6. Asia-Pacific Pompe Disease Market by Route of Administration
8.4.7. Asia-Pacific Pompe Disease Market by Dosage Forms

8.8. LAMEA

8.8.1. Key market trends
8.8.2. Key growth factors and opportunities
8.8.3. Market size and forecast

8.8.3.1. Brazil market size and forecast
8.8.3.2. Saudi Arabia market size and forecast
8.8.3.3. Argentina market size and forecast
8.8.3.4. South Africa market size and forecast
8.8.3.8. Rest of LAMEA market size and forecast

8.8.4. LAMEA Pompe Disease Market by Therapy Type
8.8.5. LAMEA Pompe Disease Market by Molecule Type
8.8.6. LAMEA Pompe Disease Market by Route of Administration
8.8.7. LAMEA Pompe Disease Market by Dosage Forms

Chapter: 9 COMPANY PROFILES

9.1. AMICUS THERAPEUTICS

9.1.1. Operating business segments
9.1.2. Business performance
9.1.3. Key strategic moves and developments

9.2. BIOMARIN PHARMACEUTICAL

9.2.1. Operating business segments
9.2.2. Business performance
9.2.3. Key strategic moves and developments

9.3. GENZYME

9.3.1. Operating business segments
9.3.2. Business performance
9.3.3. Key strategic moves and developments

9.4. AUDENTES THERAPEUTICS

9.4.1. Operating business segments
9.4.2. Business performance
9.4.3. Key strategic moves and developments

9.8. EPIVAX

9.8.1. Operating business segments
9.8.2. Business performance
9.8.3. Key strategic moves and developments

9.6. OXYRANE

9.6.1. Operating business segments
9.6.2. Business performance
9.6.3. Key strategic moves and developments

9.7. SANGAMO BIOSCIENCES

9.7.1. Operating business segments
9.7.2. Business performance
9.7.3. Key strategic moves and developments

9.8. VALERION THERAPEUTICS

9.8.1. Operating business segments
9.8.2. Business performance
9.8.3. Key strategic moves and developments

With collective industry experience of about 200 years of its analysts and experts, Allied Market Research (AMR) encompasses most infallible research methodology for its market intelligence and industry analysis. We do not only engrave the deepest levels of markets but also sneak through its slimmest details for the purpose of our market estimates and forecasts. Our approach helps in building greater market consensus view for size, shape and industry trends within each industry segment. We carefully factor in industry trends and real developments for identifying key growth factors and future course of the market. Our research proceeds are the resultant of high quality data, expert views and analysis and high value independent opinions. Our research process is designed to deliver balanced view of the global markets and allow stakeholders to make informed decisions.

We offer our clients exhaustive research and analysis based on wide variety of factual inputs, which largely include interviews with industry participants, reliable statistics and regional intelligence. Our in-house industry experts play instrumental role in designing analytic tools and models, tailored to the requirements of particular industry segment. These analytical tools and models sanitize the data & statistics and enhance the accuracy of our recommendations and advice. With AMR’s calibrated research process and 360` degree data-evaluation methodology, our clients are assured of receiving:

Consistent, valuable, robust and actionable data & analysis that can easily be referenced for strategic business planning
Technologically sophisticated and reliable insights through well audited and veracious research methodology
Sovereign research proceeds that present a tangible depiction of marketplace

With a strong methodology we are, therefore, confident that our research and analysis are most reliable and guarantees sound business planning.

Secondary research
We refer a broad array of industry sources for our secondary, which typically include; however, not limited to: Company SEC filings, annual reports, company websites, broker & financial reports and investor presentations for competitive scenario and shape of the industry

Patent and regulatory databases for understanding of technical & legal developments
Scientific and technical writings for product information and related preemptions
Regional government and statistical databases for macro analysis
Authentic new articles, web-casts and other related releases for market evaluation
Internal and external proprietary databases, key market indicators and relevant press releases for market estimates and forecast

Primary research
Our primary research efforts include reaching out participants through mail, tele-conversations, referrals, professional networks and face-to-face interactions. We are also in professional corporate relations with various companies that allow us greater flexibility for reaching out industry participants and commentators for interviews and discussions, fulfilling following functions:

Validates and improves the data quality and strengthens research proceeds
Further develops analyst team’s market understanding and expertise
Supplies authentic information about market size, share, growth and forecasts

Our primary research interview and discussion panels are typically composed of most experienced industry members. These participants include; however, not limited to:

Chief executives and VPs of leading corporations specific to industry
Product and sales managers or country heads; channel partners and top level distributors; banking, investments and valuation experts Key opinion leaders (KOLs)

Analyst tools and models
AMR has developed set of analyst tools and data models to supplement and expedite the analysis process. Corresponding to markets, where there is significant lack of information and estimates, AMR’s team of experts and analyst develop specific analyst tools and industry models to translate qualitative and quantitative industry indicators into exact industry estimates. These models also allow analysts to examine the prospects and opportunities prevailing in the market to accurately forecast the course of the market.

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Pompe Disease Market by Therapy Type (Enzyme Replacement Therapy and Substrate Reduction Therapy), Molecule Type (Biologics and Small Molecules), Route of Administration (Oral and Parenteral), and Dosage Forms (Solid and Liquid) - Global Opportunity Analysis and Industry Forecast, 2017-2023

Pompe Disease Market Key Segments:

By Therapy Type

By Molecule Type

By Route of Administration

By Dosage Forms

By Region

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Pompe Disease Market by Therapy Type (Enzyme Replacement Therapy and Substrate Reduction Therapy), Molecule Type (Biologics and Small Molecules), Route of Administration (Oral and Parenteral), and Dosage Forms (Solid and Liquid) - Global Opportunity Analysis and Industry Forecast, 2017-2023

Pompe Disease Market Key Segments:

By Therapy Type

By Molecule Type

By Route of Administration

By Dosage Forms

By Region

PURCHASE OPTIONS

Reach out to us

Request TOC/Sample

Request Customization

Request TOC/Sample

Purchase Full Report of Pompe Disease Market- Global Opportunity Analysis and Industry Forecast, 2017-2023

Why Allied Market Research

Infallible Methodology

Analyst Support

Customization

TARGETED MARKET VIEW

Featured Reports

Featured Readings

Get Fresh content delivered

Purchase Full Report of
Pompe Disease Market- Global Opportunity Analysis and Industry Forecast, 2017-2023