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 Disease Type
3.2. Snippet by Treatment Type
3.3. Snippet by End User
3.4. Snippet by Region
4. Dynamics
4.1. Impacting Factors
4.1.1. Driver
4.1.1.1. Increasing disease approvals by regulatory authorities for better treatment outcomes
4.1.1.2. Rise in the technology advancements
4.1.2. Restraints
4.1.2.1. High cost of treatment
4.1.3. Opportunity
4.1.4. Impact Analysis
5. Industry Analysis
5.1. Porter’s Five Force Analysis
5.2. Supply Chain Analysis
5.3. Pricing Analysis
5.4. Regulatory Analysis
5.5. Russia-Ukraine War Impact Analysis
5.6. DMI Opinion
6. COVID-19 Analysis
6.1. Analysis of COVID-19
6.1.1. Scenario Before COVID
6.1.2. Scenario During COVID
6.1.3. Scenario Post COVID
6.2. Pricing Dynamics Amid COVID-19
6.3. Demand-Supply Spectrum
6.4. Government Initiatives Related to the Market During Pandemic
6.5. Manufacturers Strategic Initiatives
6.6. Conclusion
7. By Disease Type
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
7.1.2. Market Attractiveness Index, By Disease Type
7.2. MPS Type 1 ((Hurler syndrome) *
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.3. MPS Type 2 (Hunter syndrome)
7.4. MPS Type 3 (Sanfilippo syndrome)
7.5. MPS Type 4 (Morquio syndrome)
7.6. MPS Type 6 (Maroteaux-Lamy syndrome)
7.7. MPS Type 7 (Sly syndrome)
7.8. MPS Type 9 (Natowicz syndrome)
8. By Treatment Type
8.1. Introduction
8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
8.1.2. Market Attractiveness Index, By Treatment Type
8.2. Enzyme Replacement Therapy *
8.2.1. Introduction
8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
8.3. Haematopoietic Stem Cell Transplantation (HSCT)
8.4. Gene Therapy
8.5. Others
9. By End User
9.1. Introduction
9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
9.1.2. Market Attractiveness Index, By End User
9.2. Hospitals *
9.2.1. Introduction
9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
9.3. Infusion Centers
9.4. Ambulatory Surgical Centers
10. By Region
10.1. Introduction
10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
10.1.2. Market Attractiveness Index, By Region
10.2. North America
10.2.1. Introduction
10.2.2. Key Region-Specific Dynamics
10.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
10.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
10.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
10.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.2.6.1. U.S.
10.2.6.2. Canada
10.2.6.3. Mexico
10.3. Europe
10.3.1. Introduction
10.3.2. Key Region-Specific Dynamics
10.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
10.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
10.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
10.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.3.6.1. Germany
10.3.6.2. UK
10.3.6.3. France
10.3.6.4. Italy
10.3.6.5. Spain
10.3.6.6. Rest of Europe
10.4. South America
10.4.1. Introduction
10.4.2. Key Region-Specific Dynamics
10.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
10.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
10.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
10.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.4.6.1. Brazil
10.4.6.2. Argentina
10.4.6.3. Rest of South America
10.5. Asia-Pacific
10.5.1. Introduction
10.5.2. Key Region-Specific Dynamics
10.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
10.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
10.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
10.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.5.6.1. China
10.5.6.2. India
10.5.6.3. Japan
10.5.6.4. Australia
10.5.6.5. Rest of Asia-Pacific
10.6. Middle East and Africa
10.6.1. Introduction
10.6.2. Key Region-Specific Dynamics
10.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
10.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
10.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
11. Competitive Landscape
11.1. Competitive Scenario
11.2. Market Positioning/Share Analysis
11.3. Mergers and Acquisitions Analysis
12. Company Profiles
12.1. BioMarin Pharmaceuticals
12.1.1. Company Overview
12.1.2. Product Portfolio and Description
12.1.3. Financial Overview
12.1.4. Key Developments
12.2. CanbridgePharma
12.3. Cerezyme
12.4. Leadiant Biosciences
12.5. Lysogene
12.6. Denali Therapeutics
12.7. Regenxbio
12.8. JCR Pharmaceuticals
12.9. Orchard Therapeutics
12.10. Inventiva (*LIST NOT EXHAUSTIVE)
13. Appendix
13.1. About Us and Services
13.2. Contact Us