Table of Content

1. Methodology and Scope
1.1. Research Methodology
1.2. Research Objective and Scope of the Report
2. Definition and Overview
3. Executive Summary
3.1. Snippet by Treatment Type
3.2. Snippet by Age Group
3.3. Snippet by End User
3.4. Snippet by Region
4. Dynamics
4.1. Impacting Factors
4.1.1. Drivers
4.1.1.1. Growing Adoption of Enzyme Replacement Therapy (ERT)
4.1.1.2. XX
4.1.2. Restraints
4.1.2.1. Limited Availability of Treatment Options in Developing Regions
4.1.3. Opportunity
4.1.4. Impact Analysis
5. Strategic Insights and Industry Outlook
5.1. Market Leaders and Pioneers
5.1.1. Emerging Pioneers and Prominent Players
5.1.2. Established leaders with largest selling Brand
5.1.3. Market leaders with established Product
5.2. CXO Perspectives
5.3. Latest Developments and Breakthroughs
5.4. Case Studies/Ongoing Research
5.5. Regulatory and Reimbursement Landscape
5.5.1. North America
5.5.2. Europe
5.5.3. Asia Pacific
5.5.4. Latin America
5.5.5. Middle East & Africa
5.6. Porter’s Five Forces Analysis
5.7. Supply Chain Analysis
5.8. Patent Analysis
5.9. SWOT Analysis
5.10. Unmet Needs and Gaps
5.11. Recommended Strategies for Market Entry and Expansion
5.12. Scenario Analysis: Best-Case, Base-Case, and Worst-Case Forecasts
5.13. Pricing Analysis and Price Dynamics
5.14. Key Opinion Leaders
6. By Treatment Type
6.1. Introduction
6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
6.1.2. Market Attractiveness Index, By Treatment Type
6.2. Enzyme Replacement Therapy (ERT)*
6.2.1. Introduction
6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
6.3. Hematopoietic Stem Cell Transplant (HSCT)
6.4. Bone Marrow Transplantation (BMT)
6.5. Others
7. By Age Group
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
7.1.2. Market Attractiveness Index, By Age Group
7.2. Adult*
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.3. Pediatric
8. By End User
8.1. Introduction
8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
8.1.2. Market Attractiveness Index, By End User
8.2. Hospitals*
8.2.1. Introduction
8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
8.3. Specialty Clinics
8.4. Others
9. By Region
9.1. Introduction
9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
9.1.2. Market Attractiveness Index, By Region
9.2. North America
9.2.1. Introduction
9.2.2. Key Region-Specific Dynamics
9.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
9.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
9.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
9.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
9.2.6.1. U.S.
9.2.6.2. Canada
9.2.6.3. Mexico
9.3. Europe
9.3.1. Introduction
9.3.2. Key Region-Specific Dynamics
9.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
9.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
9.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
9.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
9.3.6.1. Germany
9.3.6.2. U.K.
9.3.6.3. France
9.3.6.4. Spain
9.3.6.5. Italy
9.3.6.6. Rest of Europe
9.4. South America
9.4.1. Introduction
9.4.2. Key Region-Specific Dynamics
9.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
9.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
9.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
9.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
9.4.6.1. Brazil
9.4.6.2. Argentina
9.4.6.3. Rest of South America
9.5. Asia-Pacific
9.5.1. Introduction
9.5.2. Key Region-Specific Dynamics
9.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
9.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
9.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
9.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
9.5.6.1. China
9.5.6.2. India
9.5.6.3. Japan
9.5.6.4. South Korea
9.5.6.5. Rest of Asia-Pacific
9.6. Middle East and Africa
9.6.1. Introduction
9.6.2. Key Region-Specific Dynamics
9.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
9.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
9.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
10. Competitive Landscape
10.1. Competitive Scenario
10.2. Market Positioning/Share Analysis
10.3. Mergers and Acquisitions Analysis
11. Company Profiles
Key Market Players
11.1. BioMarin*
11.1.1. Company Overview
11.1.2. Product Portfolio
11.1.2.1. Product Description
11.1.2.2. Product Key Performance Indicators (KPIs)
11.1.2.3. Historic and Forecasted Product Sales
11.1.2.4. Product Sales Volume
11.1.3. Financial Overview
11.1.3.1. Company Revenue’s
11.1.3.2. Geographical Revenue Shares
11.1.3.3. Revenue Forecasts
11.1.4. Key Developments
11.1.4.1. Mergers & Acquisitions
11.1.4.2. Key Product Development Activities
11.1.4.3. Regulatory Approvals, etc.
11.1.5. SWOT Analysis
11.2. Sanofi
* Similar data will be provided for each market player.
Emerging Market Players
11.3. Orchard Therapeutics*
11.3.1. Pipeline Products Description
11.3.2. Product Key Performance Indicators (KPIs)
11.3.3. Key Activities
11.3.4. Market Entry Timelines
11.3.5. Product Penetration Rate
11.3.6. Sales Estimation and Projections
11.4. JCR Pharmaceuticals
11.5. Immusoft
11.6. Regenxbio
11.7. Paradigm Biopharmaceuticals Ltd
* Similar data will be provided for each market player.
LIST NOT EXHAUSTIVE
12. Appendix
12.1. About Us and Services
12.2. Contact Us