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Enzyme Replacement Therapy Market: Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 2024-2031
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- È¿¼Òº¸Ãæ¿ä¹ý(ERT) ½ÃÀå ±Ô¸ð(2024³â) : 110¾ï ´Þ·¯
- ½ÃÀå ±Ô¸ð ¿¹Ãø(2031³â) : 177¾ï ´Þ·¯
- ¼¼°è ½ÃÀå ¼ºÀå·ü(2024-2031³â) : CAGR 7.1%
½ÃÀå ¼ºÀå ÃËÁø¿äÀÎ :
¼¼°è È¿¼Òº¸Ãæ¿ä¹ý(ERT) ½ÃÀåÀº °í¼Åº´, ÆÄºê¸®º´, ÆûÆäº´°ú °°Àº Èñ±Í À¯ÀüÁúȯÀÇ À¯º´·ü Áõ°¡, Áø´ÜÀ² Çâ»óÀ¸·Î ÀÎÇÑ Ä¡·á ¼ö¿ä Áõ°¡ µîÀÇ ÁÖ¿ä ¿äÀο¡ ÀÇÇØ ÁÖµµµÇ°í ÀÖ½À´Ï´Ù. »ý¸í°øÇÐ, ƯÈ÷ ÀçÁ¶ÇÕ DNA ±â¼úÀÇ ¹ßÀüÀ¸·Î ¸Å¿ì È¿°úÀûÀ̰í Ç¥ÀûÈµÈ Ä¡·á¹ý °³¹ßÀÌ °¡´ÉÇØÁ³½À´Ï´Ù. ¶ÇÇÑ, Á¤ºÎÀÇ ÀçÁ¤ Áö¿ø°ú »óȯ Á¤Ã¥À¸·Î Ä¡·á Á¢±Ù¼ºÀ» ³ôÀ̰í, ±³À° Ä·ÆäÀΰú ȯÀÚ Áö¿ø ´ÜüÀÇ ÀÎ½Ä °³¼±ÀÌ Áø´ÜÀ²°ú Ä¡·á¹ý º¸±ÞÀ» ÃËÁøÇϰí ÀÖ½À´Ï´Ù.
½ÃÀå ¼ºÀå ¾ïÁ¦¿äÀÎ :
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½ÃÀå ±âȸ :
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- Sanofi Genzyme
- Shire(now part of Takeda Pharmaceuticals)
- BioMarin Pharmaceutical Inc.
- Amicus Therapeutics
- Alexion Pharmaceuticals
- Ultragenyx Pharmaceutical Inc.
- Sarepta Therapeutics
- Biomarin
- Genzyme(now part of Sanofi)
- Takeda Pharmaceuticals
- Pfizer Inc.
- AbbVie Inc.
- Vertex Pharmaceuticals
- Sangamo Therapeutics
- Regeneron Pharmaceuticals Inc.
- Spark Therapeutics(now part of Roche)
- Orchard Therapeutics
- Novartis AG
- CSL Behring
- Audentes Therapeutics
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LSH
Persistence Market Research presents an in-depth analysis of the Enzyme Replacement Therapy (ERT) Market, offering insights into its intricate dynamics and future trajectory. From market trends to growth catalysts, impediments, and emerging patterns, this report furnishes invaluable statistics and projections spanning from 2024 to 2031.
Key Insights:
- Enzyme Replacement Therapy Market Size (2024): USD 11 Bn
- Projected Market Value (2031): USD 17.7 Bn
- Global Market Growth Rate (CAGR 2024 to 2031): 7.1%
Enzyme Replacement Therapy Market - Report Scope:
The Enzyme Replacement Therapy (ERT) Market encompasses various applications across healthcare sectors such as genetic disorders, lysosomal storage diseases, and metabolic disorders. This therapy involves the replacement of deficient or absent enzymes with recombinant or synthetic alternatives, significantly improving patients' quality of life. The market caters to a wide range of end-users, including pharmaceutical companies, healthcare providers, and specialized treatment centers, offering solutions for managing chronic conditions. Market growth is driven by factors such as rising prevalence of rare diseases, increasing healthcare expenditure, and advancements in biotechnology.
Market Growth Drivers:
The global Enzyme Replacement Therapy (ERT) Market is driven by key factors, including the rising prevalence of rare genetic disorders like Gaucher's disease, Fabry disease, and Pompe disease, spurred by improved diagnosis rates that boost therapy demand. Advancements in biotechnology, particularly in recombinant DNA technology, have enabled the development of highly effective and targeted treatments. Additionally, government support through financial assistance and reimbursement policies has expanded therapy accessibility, while growing awareness fueled by educational campaigns and patient advocacy groups continues to enhance diagnosis rates and treatment adoption.
Market Restraints:
While the Enzyme Replacement Therapy (ERT) Market demonstrates significant potential, it faces notable challenges. High treatment costs, including expenses related to research, manufacturing, and distribution, limit accessibility in low- and middle-income regions. Additionally, the complex manufacturing processes required for biologically active enzymes demand stringent regulatory compliance, often slowing down commercialization. Furthermore, adverse immune reactions in some patients necessitate careful monitoring and adjustments, which can hinder broader market adoption.
Market Opportunities:
The Enzyme Replacement Therapy (ERT) Market is ripe with opportunities, propelled by advancements in research and strategic collaborations. Emerging markets like Asia-Pacific and Latin America offer significant growth potential, driven by increasing awareness and improving healthcare infrastructure. The rise of personalized medicine, facilitated by genomic research and biomarker analysis, is enabling tailored ERT solutions for enhanced efficacy. Strategic partnerships among pharmaceutical companies, research institutions, and advocacy groups are accelerating innovation and expanding the reach of enzyme therapies. Moreover, the development of oral enzyme formulations and gene therapies marks a transformative shift, potentially replacing traditional intravenous treatments.
Key Questions Answered in the Report:
- What are the primary factors driving the growth of the Enzyme Replacement Therapy Market globally?
- How are technological advancements and innovative delivery systems shaping the evolution of enzyme therapies?
- What are the challenges and opportunities facing market participants in the ERT segment?
- Which therapeutic areas and geographic regions offer the highest growth potential for enzyme replacement therapy manufacturers?
- What strategies are leading companies employing to differentiate their offerings and capture market share in the competitive landscape?
Competitive Intelligence and Business Strategy:
Leading players in the global Enzyme Replacement Therapy Market are prioritizing innovation, strategic partnerships, and market expansion to secure a competitive edge. Companies are investing heavily in research and development to improve the efficacy and safety of enzyme therapies. Collaboration with academic institutions and patient advocacy organizations facilitates access to cutting-edge research and fosters community support.
Additionally, manufacturers are focusing on patient-centric initiatives, such as affordability programs and educational campaigns, to increase awareness and adoption of enzyme replacement therapies. Emphasis on quality assurance, regulatory compliance, and post-marketing surveillance enhances trust and long-term relationships with healthcare providers and patients.
Key Companies Profiled:
- Sanofi S.A.
- Pfizer Inc.
- Shire (a Takeda company)
- Biomarin Pharmaceutical Inc.
- Alexion Pharmaceuticals, Inc.
- AbbVie Inc.
- Amicus Therapeutics, Inc.
- Roche Holding AG
- CSL Behring
- Horizon Therapeutics Plc
- Genzyme Corporation
Enzyme Replacement Therapy Market Research Segmentation:
By Therapeutic Area:
- Lysosomal Storage Disorders
- Gaucher Disease
- Fabry Disease
- Pompe Disease
- Mucopolysaccharidosis
- Metabolic Disorders
- Others
By Route of Administration:
By End-User:
- Hospitals
- Specialty Clinics
- Research Institutes
By Region
North America
Latin America
Europe
South Asia and Oceania
East Asia
Middle East and Africa
Table of Contents
1. Executive Summary
- 1.1. Global Enzyme Replacement Therapy Market Snapshot, 2024 and 2031
- 1.2. Market Opportunity Assessment, 2024 - 2031, US$ Bn
- 1.3. Key Market Trends
- 1.4. Future Market Projections
- 1.5. Premium Market Insights
- 1.6. Industry Developments and Key Market Events
- 1.7. PMR Analysis and Recommendations
2. Market Overview
- 2.1. Market Scope and Definition
- 2.2. Market Dynamics
- 2.2.1. Drivers
- 2.2.2. Restraints
- 2.2.3. Opportunity
- 2.2.4. Challenges
- 2.2.5. Key Trends
- 2.3. Macro-Economic Factors
- 2.3.1. Global Sectorial Outlook
- 2.3.2. Global GDP Growth Outlook
- 2.3.3. Global Healthcare Spending Outlook
- 2.4. COVID-19 Impact Analysis
- 2.5. Forecast Factors - Relevance and Impact
3. Value Added Insights
- 3.1. Therapeutic Conditions Adoption Analysis
- 3.2. Technology Assessment
- 3.3. Regulatory Landscape
- 3.4. Value Chain Analysis
- 3.5. Key Deals and Mergers
- 3.6. PESTLE Analysis
- 3.7. Porter's Five Force Analysis
4. Price Trend Analysis, 2019 - 2031
- 4.1. Key Highlights
- 4.2. Key Factors Impacting Product Prices
- 4.3. Pricing Analysis, By Therapeutic Conditions
- 4.4. Regional Prices and Product Preferences
5. Global Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
- 5.1. Key Highlights
- 5.1.1. Market Volume (Units) Projections
- 5.1.2. Market Size (US$ Bn) and Volume (Units) and Y-o-Y Growth
- 5.1.3. Absolute $ Opportunity
- 5.2. Market Size (US$ Bn) and Volume (Units) Analysis and Forecast
- 5.2.1. Historical Market Size (US$ Bn) and Volume (Units) Analysis, 2019 - 2023
- 5.2.2. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, 2023-2031
- 5.3. Global Enzyme Replacement Therapy Market Outlook: Therapeutic Conditions
- 5.3.1. Introduction / Key Findings
- 5.3.2. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Therapeutic Conditions, 2019 - 2023
- 5.3.3. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
- 5.3.3.1. Fabry Disease
- 5.3.3.2. Gaucher Disease
- 5.3.3.3. Mucopolysaccharidosis (MPS)
- 5.3.3.3.1. MPS I (Hurler Syndrome)
- 5.3.3.3.2. MPS II (Hunter Syndrome)
- 5.3.3.3.3. MPS IVA (Morquio Syndrome, Type A)
- 5.3.3.3.4. MPS VI (Maroteaux-Lamy Syndrome)
- 5.3.3.3.5. MPS VII (Sly Syndrome)
- 5.3.3.4. Pompe Disease
- 5.3.3.5. Lysosomal Acid Lipase Deficiency
- 5.3.3.6. Others
- 5.4. Market Attractiveness Analysis: Therapeutic Conditions
- 5.5. Global Enzyme Replacement Therapy Market Outlook: Route of Administration
- 5.5.1. Introduction / Key Findings
- 5.5.2. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Route of Administration, 2019 - 2023
- 5.5.3. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
- 5.5.3.1. Oral
- 5.5.3.2. Injectable
- 5.6. Market Attractiveness Analysis: Route of Administration
- 5.7. Global Enzyme Replacement Therapy Market Outlook: Distribution Channel
- 5.7.1. Introduction / Key Findings
- 5.7.2. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Distribution Channel, 2019 - 2023
- 5.7.3. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
- 5.7.3.1. Hospital Pharmacies
- 5.7.3.2. Specialty Treatment Pharmacies
- 5.7.3.3. Retail Pharmacies
- 5.8. Market Attractiveness Analysis: Distribution Channel
6. Global Enzyme Replacement Therapy Market Outlook: Region
- 6.1. Key Highlights
- 6.2. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Region, 2019 - 2023
- 6.3. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Region, 2024 - 2031
- 6.3.1. North America
- 6.3.2. Europe
- 6.3.3. East Asia
- 6.3.4. South Asia and Oceania
- 6.3.5. Latin America
- 6.3.6. Middle East & Africa
- 6.4. Market Attractiveness Analysis: Region
7. North America Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
- 7.1. Key Highlights
- 7.2. Pricing Analysis
- 7.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
- 7.3.1. By Country
- 7.3.2. By Therapeutic Conditions
- 7.3.3. By Route of Administration
- 7.3.4. By Distribution Channel
- 7.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
- 7.4.1. U.S.
- 7.4.2. Canada
- 7.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
- 7.5.1. Enzyme Replacement Therapy for Fabry Disease
- 7.5.2. Enzyme Replacement Therapy for Gaucher Disease
- 7.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
- 7.5.4. Enzyme Replacement Therapy for Pompe Disease
- 7.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
- 7.5.6. Others
- 7.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
- 7.6.1. Oral Enzyme Replacement Therapy
- 7.6.2. Injectable Enzyme Replacement Therapy
- 7.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
- 7.7.1. Hospital Pharmacies
- 7.7.2. Specialty Treatment Pharmacies
- 7.7.3. Retail Pharmacies
- 7.8. Market Attractiveness Analysis
8. Europe Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
- 8.1. Key Highlights
- 8.2. Pricing Analysis
- 8.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
- 8.3.1. By Country
- 8.3.2. By Therapeutic Conditions
- 8.3.3. By Route of Administration
- 8.3.4. By Distribution Channel
- 8.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
- 8.4.1. Germany
- 8.4.2. France
- 8.4.3. U.K.
- 8.4.4. Italy
- 8.4.5. Spain
- 8.4.6. Russia
- 8.4.7. Turkey
- 8.4.8. Rest of Europe
- 8.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
- 8.5.1. Enzyme Replacement Therapy for Fabry Disease
- 8.5.2. Enzyme Replacement Therapy for Gaucher Disease
- 8.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
- 8.5.4. Enzyme Replacement Therapy for Pompe Disease
- 8.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
- 8.5.6. Others
- 8.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
- 8.6.1. Oral Enzyme Replacement Therapy
- 8.6.2. Injectable Enzyme Replacement Therapy
- 8.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
- 8.7.1. Hospital Pharmacies
- 8.7.2. Specialty Treatment Pharmacies
- 8.7.3. Retail Pharmacies
- 8.8. Market Attractiveness Analysis
9. East Asia Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
- 9.1. Key Highlights
- 9.2. Pricing Analysis
- 9.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
- 9.3.1. By Country
- 9.3.2. By Therapeutic Conditions
- 9.3.3. By Route of Administration
- 9.3.4. By Distribution Channel
- 9.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
- 9.4.1. China
- 9.4.2. Japan
- 9.4.3. South Korea
- 9.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
- 9.5.1. Enzyme Replacement Therapy for Fabry Disease
- 9.5.2. Enzyme Replacement Therapy for Gaucher Disease
- 9.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
- 9.5.4. Enzyme Replacement Therapy for Pompe Disease
- 9.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
- 9.5.6. Others
- 9.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
- 9.6.1. Oral Enzyme Replacement Therapy
- 9.6.2. Injectable Enzyme Replacement Therapy
- 9.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
- 9.7.1. Hospital Pharmacies
- 9.7.2. Specialty Treatment Pharmacies
- 9.7.3. Retail Pharmacies
- 9.8. Market Attractiveness Analysis
10. South Asia & Oceania Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
- 10.1. Key Highlights
- 10.2. Pricing Analysis
- 10.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
- 10.3.1. By Country
- 10.3.2. By Therapeutic Conditions
- 10.3.3. By Route of Administration
- 10.3.4. By Distribution Channel
- 10.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
- 10.4.1. India
- 10.4.2. Southeast Asia
- 10.4.3. ANZ
- 10.4.4. Rest of South Asia & Oceania
- 10.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
- 10.5.1. Enzyme Replacement Therapy for Fabry Disease
- 10.5.2. Enzyme Replacement Therapy for Gaucher Disease
- 10.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
- 10.5.4. Enzyme Replacement Therapy for Pompe Disease
- 10.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
- 10.5.6. Others
- 10.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
- 10.6.1. Oral Enzyme Replacement Therapy
- 10.6.2. Injectable Enzyme Replacement Therapy
- 10.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
- 10.7.1. Hospital Pharmacies
- 10.7.2. Specialty Treatment Pharmacies
- 10.7.3. Retail Pharmacies
- 10.8. Market Attractiveness Analysis
11. Latin America Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
- 11.1. Key Highlights
- 11.2. Pricing Analysis
- 11.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
- 11.3.1. By Country
- 11.3.2. By Therapeutic Conditions
- 11.3.3. By Route of Administration
- 11.3.4. By Distribution Channel
- 11.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
- 11.4.1. Brazil
- 11.4.2. Mexico
- 11.4.3. Rest of Latin America
- 11.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
- 11.5.1. Enzyme Replacement Therapy for Fabry Disease
- 11.5.2. Enzyme Replacement Therapy for Gaucher Disease
- 11.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
- 11.5.4. Enzyme Replacement Therapy for Pompe Disease
- 11.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
- 11.5.6. Others
- 11.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
- 11.6.1. Oral Enzyme Replacement Therapy
- 11.6.2. Injectable Enzyme Replacement Therapy
- 11.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
- 11.7.1. Hospital Pharmacies
- 11.7.2. Specialty Treatment Pharmacies
- 11.7.3. Retail Pharmacies
- 11.8. Market Attractiveness Analysis
12. Middle East & Africa Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
- 12.1. Key Highlights
- 12.2. Pricing Analysis
- 12.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
- 12.3.1. By Country
- 12.3.2. By Therapeutic Conditions
- 12.3.3. By Route of Administration
- 12.3.4. By Distribution Channel
- 12.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
- 12.4.1. GCC Countries
- 12.4.2. Egypt
- 12.4.3. South Africa
- 12.4.4. Northern Africa
- 12.4.5. Rest of Middle East & Africa
- 12.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
- 12.5.1. Enzyme Replacement Therapy for Fabry Disease
- 12.5.2. Enzyme Replacement Therapy for Gaucher Disease
- 12.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
- 12.5.4. Enzyme Replacement Therapy for Pompe Disease
- 12.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
- 12.5.6. Others
- 12.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
- 12.6.1. Oral Enzyme Replacement Therapy
- 12.6.2. Injectable Enzyme Replacement Therapy
- 12.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
- 12.7.1. Hospital Pharmacies
- 12.7.2. Specialty Treatment Pharmacies
- 12.7.3. Retail Pharmacies
- 12.8. Market Attractiveness Analysis
13. Competition Landscape
- 13.1. Market Share Analysis, 2023
- 13.2. Market Structure
- 13.2.1. Competition Intensity Mapping By Market
- 13.2.2. Competition Dashboard
- 13.3. Company Profiles (Details - Overview, Financials, Strategy, Recent Developments)
- 13.3.1. Sanofi Genzyme
- 13.3.1.1. Overview
- 13.3.1.2. Segments and Therapeutic Conditions
- 13.3.1.3. Key Financials
- 13.3.1.4. Market Developments
- 13.3.1.5. Market Strategy
- 13.3.2. Shire (now part of Takeda Pharmaceuticals)
- 13.3.3. BioMarin Pharmaceutical Inc.
- 13.3.4. Amicus Therapeutics
- 13.3.5. Alexion Pharmaceuticals
- 13.3.6. Ultragenyx Pharmaceutical Inc.
- 13.3.7. Sarepta Therapeutics
- 13.3.8. Biomarin
- 13.3.9. Genzyme (now part of Sanofi)
- 13.3.10. Takeda Pharmaceuticals
- 13.3.11. Pfizer Inc.
- 13.3.12. AbbVie Inc.
- 13.3.13. Vertex Pharmaceuticals
- 13.3.14. Sangamo Therapeutics
- 13.3.15. Regeneron Pharmaceuticals Inc.
- 13.3.16. Spark Therapeutics (now part of Roche)
- 13.3.17. Orchard Therapeutics
- 13.3.18. Novartis AG
- 13.3.19. CSL Behring
- 13.3.20. Audentes Therapeutics
14. Appendix
- 14.1. Research Methodology
- 14.2. Research Assumptions
- 14.3. Acronyms and Abbreviations