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 Type
3.2. Snippet by Treatment Type
3.3. Snippet by Age Group
3.4. Snippet by End User
4. Dynamics
4.1. Impacting Factors
4.1.1. Drivers
4.1.1.1. Rising innovations in products and product approvals
4.1.1.2. Rising advances in diagnostic procedures
4.1.2. Restraints
4.1.2.1. High cost associated with the 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. Unmet Needs
5.6. PESTEL Analysis
5.7. Patent Analysis
5.8. SWOT Analysis
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 Type
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
7.1.2. Market Attractiveness Index, By Type
7.2. Phenylketonuria*
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.3. Adrenoleukodystrophy
7.4. Gaucher’s Disease
7.5. Fabre Disease
7.6. Pompe Disease
7.7. Others
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. Cofactor Replacement Therapy
8.4. Gene Therapy
8.5. Vitamin Therapy
8.6. Others
9. By Age Group
9.1. Introduction
9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
9.1.2. Market Attractiveness Index, By Age Group
9.2. Pediatrics*
9.2.1. Introduction
9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
9.3. Adults
10. By End Users
10.1. Introduction
10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End Users
10.1.2. Market Attractiveness Index, By End Users
10.2. Hospitals & Clinics*
10.2.1. Introduction
10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
10.3. Pediatric Care Centers
10.4. Specialty Clinics
10.5. Homecare
10.6. Others
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 Pharmaceutical Inc.
12.1.1. Company Overview
12.1.2. Product Portfolio and Description
12.1.3. Financial Overview
12.1.4. Key Developments
12.2. Sanofi
12.3. Takeda Pharmaceuticals U.S.A., Inc.
12.4. Pfizer Inc.
12.5. Actelion Pharmaceuticals US, Inc.
12.6. Bluebird Bio, Inc.
12.7. Orchard Therapeutics plc
12.8. Amicus Therapeutics, Inc.
12.9. CHIESI Farmaceutici S.p.A.
12.10. Sentynl Therapeutics, Inc.
LIST NOT EXHAUSTIVE
13. Appendix
13.1. About Us and Services
13.2. Contact Us