The "Global Gene Therapy in CNS Disorder Market" was valued at US$ 250.00 million in 2024 and is predicted to reach US$ 621.81 million by 2030, exhibiting a Compound Annual Growth Rate (CAGR) of 16.4% during the forecast period (2024-2030).
Gene therapy for central nervous system (CNS) disorders involves the introduction, modification, or replacement of genes within the brain and spinal cord to treat or prevent neurological diseases. CNS disorders such as Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, and various genetic or neurodegenerative conditions have complex pathologies, and gene therapy offers a promising approach by directly targeting the underlying genetic causes of these disorders.
Key components of gene therapy in CNS disorders:
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Mechanism:
- Gene therapy typically works by delivering a therapeutic gene to the cells of the CNS using a vector, usually a virus (such as an adeno-associated virus, or AAV). This can either replace a faulty gene, introduce a new gene to help the cells function better, or silence a gene that is causing harmful effects.
- Delivery methods include:
- Direct injection into the brain or spinal cord (intracerebral or intrathecal administration).
- Intravenous (IV) administration where the therapeutic gene is carried through the bloodstream to the CNS.
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Targets and Strategies:
- Gene replacement: Used for genetic disorders like spinal muscular atrophy (SMA) where a defective gene is replaced with a functional copy.
- Gene silencing: Techniques like RNA interference (RNAi) or CRISPR can silence harmful genes, as in Huntington’s disease where abnormal proteins are reduced.
- Neuroprotection: Introducing genes that code for protective proteins to slow or stop the degeneration of neurons, which is useful in diseases like Parkinson’s.
- Anti-inflammatory strategies: Gene therapy may also be used to reduce inflammation that contributes to CNS damage, such as in multiple sclerosis (MS).
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Advantages:
- Targeting the root cause: Unlike traditional therapies that address symptoms, gene therapy directly modifies the underlying genetic or molecular pathways causing the disease.
- Long-term effects: Gene therapy can potentially offer long-lasting or permanent treatment effects after a single administration, reducing the need for continuous medication.
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Challenges:
- Delivery difficulties: The blood-brain barrier (BBB) makes it difficult for therapeutic agents to reach the CNS, requiring invasive methods or specialized delivery systems.
- Safety concerns: As with any genetic modification, there are risks related to immune reactions, off-target effects, and long-term consequences.
- Complexity of CNS diseases: Many CNS disorders are caused by multiple genes or environmental factors, making them more challenging to treat with a single gene therapy.
Applications in CNS disorders:
- Spinal Muscular Atrophy (SMA): Gene therapy, like Zolgensma, replaces the faulty SMN1 gene, allowing for improved motor function and survival in affected infants.
- Parkinson’s Disease: Gene therapy aims to increase the production of dopamine or neuroprotective factors to slow the disease progression.
- Huntington’s Disease: Gene silencing therapies are being developed to reduce the production of harmful mutant proteins.
- Alzheimer’s Disease: Gene therapy approaches are being explored to reduce amyloid-beta plaques or improve neurogenesis.
Gene therapy holds great potential in transforming the treatment landscape for CNS disorders, providing hope for addressing currently untreatable or poorly managed conditions.
The global key manufacturers of Gene Therapy in CNS Disorder include Voyager Therapeutics, Spark Therapeutics, Novartis AG, Bluebird Bio Inc. and Biogen, etc. in 2023, the global top five players have a share approximately % in terms of revenue.
Gene therapyentails the specific transfer of genetic information into affected cells and tissues in order to restore function in diseased areas.
This report aims to provide a comprehensive presentation of the global market for Gene Therapy in CNS Disorder, with both quantitative and qualitative analysis, to help readers develop business/growth strategies, assess the market competitive situation, analyze their position in the current marketplace, and make informed business decisions regarding Gene Therapy in CNS Disorder. This report contains market size and forecasts of Gene Therapy in CNS Disorder in global, including the following market information:
- Global Gene Therapy in CNS Disorder Market Revenue, 2019-2024, 2025-2030, ($ millions)
- Global top five companies in 2023 (%)
We has surveyed the Gene Therapy in CNS Disorder companies, and industry experts on this industry, involving the revenue, demand, product type, recent developments and plans, industry trends, drivers, challenges, obstacles, and potential risks.
Total Market by Segment:
Global Gene Therapy in CNS Disorder Market, by Type, 2019-2024, 2025-2030 ($ millions)
Global Gene Therapy in CNS Disorder Market Segment Percentages, by Type, 2023 (%)
- Viral Gene Therapy
- Non-viral Gene Therapy
Global Gene Therapy in CNS Disorder Market, by Application, 2019-2024, 2025-2030 ($ millions)
Global Gene Therapy in CNS Disorder Market Segment Percentages, by Application, 2023 (%)
- Alzheimer's Disease
- Huntington's Disease
- Parkinson's Disease
- Batten Disease
Global Gene Therapy in CNS Disorder Market, By Region and Country, 2019-2024, 2025-2030 ($ Millions)
Global Gene Therapy in CNS Disorder Market Segment Percentages, By Region and Country, 2023 (%)
- North America (United States, Canada, Mexico)
- Europe (Germany, France, United Kingdom, Italy, Spain, Rest of Europe)
- Asia-Pacific (China, India, Japan, South Korea, Australia, Rest of APAC)
- The Middle East and Africa (Middle East, Africa)
- South and Central America (Brazil, Argentina, Rest of SCA)
Competitor Analysis
The report also provides analysis of leading market participants including:
- Key companies Gene Therapy in CNS Disorder revenues in global market, 2019-2024 (estimated), ($ millions)
- Key companies Gene Therapy in CNS Disorder revenues share in global market, 2023 (%)
Further, the report presents profiles of competitors in the market, key players include:
- Voyager Therapeutics
- Spark Therapeutics
- Novartis AG
- Bluebird Bio Inc.
- Biogen
Outline of Major Chapters:
Chapter 1: Introduces the definition of Gene Therapy in CNS Disorder, market overview.
Chapter 2: Global Gene Therapy in CNS Disorder market size in revenue.
Chapter 3: Detailed analysis of Gene Therapy in CNS Disorder company competitive landscape, revenue and market share, latest development plan, merger, and acquisition information, etc.
Chapter 4: Provides the analysis of various market segments by type, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different market segments.
Chapter 5: Provides the analysis of various market segments by application, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different downstream markets.
Chapter 6: Sales of Gene Therapy in CNS Disorder in regional level and country level. It provides a quantitative analysis of the market size and development potential of each region and its main countries and introduces the market development, future development prospects, market space of each country in the world.
Chapter 7: Provides profiles of key players, introducing the basic situation of the main companies in the market in detail, including product sales, revenue, price, gross margin, product introduction, recent development, etc.
Chapter 8: The main points and conclusions of the report.
TABLE OF CONTENTS
1 Introduction to Research & Analysis Reports
1.1 Gene Therapy in CNS Disorder Market Definition
1.2 Market Segments
1.2.1 Market by Type
1.2.2 Market by Application
1.3 Global Gene Therapy in CNS Disorder Market Overview
1.4 Features & Benefits of This Report
1.5 Methodology & Sources of Information
1.5.1 Research Methodology
1.5.2 Research Process
1.5.3 Base Year
1.5.4 Report Assumptions & Caveats
2 Global Gene Therapy in CNS Disorder Overall Market Size
2.1 Global Gene Therapy in CNS Disorder Market Size: 2023 VS 2030
2.2 Global Gene Therapy in CNS Disorder Market Size, Prospects & Forecasts: 2019-2030
2.3 Key Market Trends, Opportunity, Drivers and Restraints
2.3.1 Market Opportunities & Trends
2.3.2 Market Drivers
2.3.3 Market Restraints
3 Company Landscape
3.1 Top Gene Therapy in CNS Disorder Players in Global Market
3.2 Top Global Gene Therapy in CNS Disorder Companies Ranked by Revenue
3.3 Global Gene Therapy in CNS Disorder Revenue by Companies
3.4 Top 3 and Top 5 Gene Therapy in CNS Disorder Companies in Global Market, by Revenue in 2023
3.5 Global Companies Gene Therapy in CNS Disorder Product Type
3.6 Tier 1, Tier 2 and Tier 3 Gene Therapy in CNS Disorder Players in Global Market
3.6.1 List of Global Tier 1 Gene Therapy in CNS Disorder Companies
3.6.2 List of Global Tier 2 and Tier 3 Gene Therapy in CNS Disorder Companies
4 Market Sights by Product
4.1 Overview
4.1.1 By Type - Global Gene Therapy in CNS Disorder Market Size Markets, 2023 & 2030
4.1.2 Viral Gene Therapy
4.1.3 Non-viral Gene Therapy
4.2 By Type - Global Gene Therapy in CNS Disorder Revenue & Forecasts
4.2.1 By Type - Global Gene Therapy in CNS Disorder Revenue, 2019-2024
4.2.2 By Type - Global Gene Therapy in CNS Disorder Revenue, 2025-2030
4.2.3 By Type - Global Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
5 Sights by Application
5.1 Overview
5.1.1 By Application - Global Gene Therapy in CNS Disorder Market Size, 2023 & 2030
5.1.2 Alzheimer's Disease
5.1.3 Huntington's Disease
5.1.4 Parkinson's Disease
5.1.5 Batten Disease
5.2 By Application - Global Gene Therapy in CNS Disorder Revenue & Forecasts
5.2.1 By Application - Global Gene Therapy in CNS Disorder Revenue, 2019-2024
5.2.2 By Application - Global Gene Therapy in CNS Disorder Revenue, 2025-2030
5.2.3 By Application - Global Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
6 Sights by Region
6.1 By Region - Global Gene Therapy in CNS Disorder Market Size, 2023 & 2030
6.2 By Region - Global Gene Therapy in CNS Disorder Revenue & Forecasts
6.2.1 By Region - Global Gene Therapy in CNS Disorder Revenue, 2019-2024
6.2.2 By Region - Global Gene Therapy in CNS Disorder Revenue, 2025-2030
6.2.3 By Region - Global Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
6.3 North America
6.3.1 By Country - North America Gene Therapy in CNS Disorder Revenue, 2019-2030
6.3.2 US Gene Therapy in CNS Disorder Market Size, 2019-2030
6.3.3 Canada Gene Therapy in CNS Disorder Market Size, 2019-2030
6.3.4 Mexico Gene Therapy in CNS Disorder Market Size, 2019-2030
6.4 Europe
6.4.1 By Country - Europe Gene Therapy in CNS Disorder Revenue, 2019-2030
6.4.2 Germany Gene Therapy in CNS Disorder Market Size, 2019-2030
6.4.3 France Gene Therapy in CNS Disorder Market Size, 2019-2030
6.4.4 U.K. Gene Therapy in CNS Disorder Market Size, 2019-2030
6.4.5 Italy Gene Therapy in CNS Disorder Market Size, 2019-2030
6.4.6 Russia Gene Therapy in CNS Disorder Market Size, 2019-2030
6.4.7 Nordic Countries Gene Therapy in CNS Disorder Market Size, 2019-2030
6.4.8 Benelux Gene Therapy in CNS Disorder Market Size, 2019-2030
6.5 Asia
6.5.1 By Region - Asia Gene Therapy in CNS Disorder Revenue, 2019-2030
6.5.2 China Gene Therapy in CNS Disorder Market Size, 2019-2030
6.5.3 Japan Gene Therapy in CNS Disorder Market Size, 2019-2030
6.5.4 South Korea Gene Therapy in CNS Disorder Market Size, 2019-2030
6.5.5 Southeast Asia Gene Therapy in CNS Disorder Market Size, 2019-2030
6.5.6 India Gene Therapy in CNS Disorder Market Size, 2019-2030
6.6 South America
6.6.1 By Country - South America Gene Therapy in CNS Disorder Revenue, 2019-2030
6.6.2 Brazil Gene Therapy in CNS Disorder Market Size, 2019-2030
6.6.3 Argentina Gene Therapy in CNS Disorder Market Size, 2019-2030
6.7 Middle East & Africa
6.7.1 By Country - Middle East & Africa Gene Therapy in CNS Disorder Revenue, 2019-2030
6.7.2 Turkey Gene Therapy in CNS Disorder Market Size, 2019-2030
6.7.3 Israel Gene Therapy in CNS Disorder Market Size, 2019-2030
6.7.4 Saudi Arabia Gene Therapy in CNS Disorder Market Size, 2019-2030
6.7.5 UAE Gene Therapy in CNS Disorder Market Size, 2019-2030
7 Gene Therapy in CNS Disorder Companies Profiles
7.1 Voyager Therapeutics
7.1.1 Voyager Therapeutics Company Summary
7.1.2 Voyager Therapeutics Business Overview
7.1.3 Voyager Therapeutics Gene Therapy in CNS Disorder Major Product Offerings
7.1.4 Voyager Therapeutics Gene Therapy in CNS Disorder Revenue in Global Market (2019-2024)
7.1.5 Voyager Therapeutics Key News & Latest Developments
7.2 Spark Therapeutics
7.2.1 Spark Therapeutics Company Summary
7.2.2 Spark Therapeutics Business Overview
7.2.3 Spark Therapeutics Gene Therapy in CNS Disorder Major Product Offerings
7.2.4 Spark Therapeutics Gene Therapy in CNS Disorder Revenue in Global Market (2019-2024)
7.2.5 Spark Therapeutics Key News & Latest Developments
7.3 Novartis AG
7.3.1 Novartis AG Company Summary
7.3.2 Novartis AG Business Overview
7.3.3 Novartis AG Gene Therapy in CNS Disorder Major Product Offerings
7.3.4 Novartis AG Gene Therapy in CNS Disorder Revenue in Global Market (2019-2024)
7.3.5 Novartis AG Key News & Latest Developments
7.4 Bluebird Bio Inc.
7.4.1 Bluebird Bio Inc. Company Summary
7.4.2 Bluebird Bio Inc. Business Overview
7.4.3 Bluebird Bio Inc. Gene Therapy in CNS Disorder Major Product Offerings
7.4.4 Bluebird Bio Inc. Gene Therapy in CNS Disorder Revenue in Global Market (2019-2024)
7.4.5 Bluebird Bio Inc. Key News & Latest Developments
7.5 Biogen
7.5.1 Biogen Company Summary
7.5.2 Biogen Business Overview
7.5.3 Biogen Gene Therapy in CNS Disorder Major Product Offerings
7.5.4 Biogen Gene Therapy in CNS Disorder Revenue in Global Market (2019-2024)
7.5.5 Biogen Key News & Latest Developments
8 Conclusion
9 Appendix
9.1 Note
9.2 Examples of Clients
9.3 Disclaimer
LIST OF TABLES & FIGURES
List of Tables
Table 1. Gene Therapy in CNS Disorder Market Opportunities & Trends in Global Market
Table 2. Gene Therapy in CNS Disorder Market Drivers in Global Market
Table 3. Gene Therapy in CNS Disorder Market Restraints in Global Market
Table 4. Key Players of Gene Therapy in CNS Disorder in Global Market
Table 5. Top Gene Therapy in CNS Disorder Players in Global Market, Ranking by Revenue (2023)
Table 6. Global Gene Therapy in CNS Disorder Revenue by Companies, (US$, Mn), 2019-2024
Table 7. Global Gene Therapy in CNS Disorder Revenue Share by Companies, 2019-2024
Table 8. Global Companies Gene Therapy in CNS Disorder Product Type
Table 9. List of Global Tier 1 Gene Therapy in CNS Disorder Companies, Revenue (US$, Mn) in 2023 and Market Share
Table 10. List of Global Tier 2 and Tier 3 Gene Therapy in CNS Disorder Companies, Revenue (US$, Mn) in 2023 and Market Share
Table 11. By Type ? Global Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2023 & 2030
Table 12. By Type - Gene Therapy in CNS Disorder Revenue in Global (US$, Mn), 2019-2024
Table 13. By Type - Gene Therapy in CNS Disorder Revenue in Global (US$, Mn), 2025-2030
Table 14. By Application ? Global Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2023 & 2030
Table 15. By Application - Gene Therapy in CNS Disorder Revenue in Global (US$, Mn), 2019-2024
Table 16. By Application - Gene Therapy in CNS Disorder Revenue in Global (US$, Mn), 2025-2030
Table 17. By Region ? Global Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2023 & 2030
Table 18. By Region - Global Gene Therapy in CNS Disorder Revenue (US$, Mn), 2019-2024
Table 19. By Region - Global Gene Therapy in CNS Disorder Revenue (US$, Mn), 2025-2030
Table 20. By Country - North America Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2024
Table 21. By Country - North America Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2025-2030
Table 22. By Country - Europe Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2024
Table 23. By Country - Europe Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2025-2030
Table 24. By Region - Asia Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2024
Table 25. By Region - Asia Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2025-2030
Table 26. By Country - South America Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2024
Table 27. By Country - South America Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2025-2030
Table 28. By Country - Middle East & Africa Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2024
Table 29. By Country - Middle East & Africa Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2025-2030
Table 30. Voyager Therapeutics Company Summary
Table 31. Voyager Therapeutics Gene Therapy in CNS Disorder Product Offerings
Table 32. Voyager Therapeutics Gene Therapy in CNS Disorder Revenue (US$, Mn) & (2019-2024)
Table 33. Voyager Therapeutics Key News & Latest Developments
Table 34. Spark Therapeutics Company Summary
Table 35. Spark Therapeutics Gene Therapy in CNS Disorder Product Offerings
Table 36. Spark Therapeutics Gene Therapy in CNS Disorder Revenue (US$, Mn) & (2019-2024)
Table 37. Spark Therapeutics Key News & Latest Developments
Table 38. Novartis AG Company Summary
Table 39. Novartis AG Gene Therapy in CNS Disorder Product Offerings
Table 40. Novartis AG Gene Therapy in CNS Disorder Revenue (US$, Mn) & (2019-2024)
Table 41. Novartis AG Key News & Latest Developments
Table 42. Bluebird Bio Inc. Company Summary
Table 43. Bluebird Bio Inc. Gene Therapy in CNS Disorder Product Offerings
Table 44. Bluebird Bio Inc. Gene Therapy in CNS Disorder Revenue (US$, Mn) & (2019-2024)
Table 45. Bluebird Bio Inc. Key News & Latest Developments
Table 46. Biogen Company Summary
Table 47. Biogen Gene Therapy in CNS Disorder Product Offerings
Table 48. Biogen Gene Therapy in CNS Disorder Revenue (US$, Mn) & (2019-2024)
Table 49. Biogen Key News & Latest Developments
List of Figures
Figure 1. Gene Therapy in CNS Disorder Segment by Type in 2023
Figure 2. Gene Therapy in CNS Disorder Segment by Application in 2023
Figure 3. Global Gene Therapy in CNS Disorder Market Overview: 2023
Figure 4. Key Caveats
Figure 5. Global Gene Therapy in CNS Disorder Market Size: 2023 VS 2030 (US$, Mn)
Figure 6. Global Gene Therapy in CNS Disorder Revenue, 2019-2030 (US$, Mn)
Figure 7. The Top 3 and 5 Players Market Share by Gene Therapy in CNS Disorder Revenue in 2023
Figure 8. By Type - Global Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
Figure 9. By Application - Global Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
Figure 10. By Type - Global Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2023 & 2030
Figure 11. By Type - Global Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
Figure 12. By Application - Global Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2023 & 2030
Figure 13. By Application - Global Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
Figure 14. By Region - Global Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
Figure 15. By Country - North America Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
Figure 16. US Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 17. Canada Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 18. Mexico Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 19. By Country - Europe Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
Figure 20. Germany Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 21. France Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 22. U.K. Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 23. Italy Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 24. Russia Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 25. Nordic Countries Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 26. Benelux Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 27. By Region - Asia Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
Figure 28. China Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 29. Japan Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 30. South Korea Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 31. Southeast Asia Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 32. India Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 33. By Country - South America Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
Figure 34. Brazil Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 35. Argentina Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 36. By Country - Middle East & Africa Gene Therapy in CNS Disorder Revenue Market Share, 2019-2030
Figure 37. Turkey Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 38. Israel Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 39. Saudi Arabia Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 40. UAE Gene Therapy in CNS Disorder Revenue, (US$, Mn), 2019-2030
Figure 41. Voyager Therapeutics Gene Therapy in CNS Disorder Revenue Year Over Year Growth (US$, Mn) & (2019-2024)
Figure 42. Spark Therapeutics Gene Therapy in CNS Disorder Revenue Year Over Year Growth (US$, Mn) & (2019-2024)
Figure 43. Novartis AG Gene Therapy in CNS Disorder Revenue Year Over Year Growth (US$, Mn) & (2019-2024)
Figure 44. Bluebird Bio Inc. Gene Therapy in CNS Disorder Revenue Year Over Year Growth (US$, Mn) & (2019-2024)
Figure 45. Biogen Gene Therapy in CNS Disorder Revenue Year Over Year Growth (US$, Mn) & (2019-2024)
