1 Introduction to Research & Analysis Reports
1.1 Spinal Muscular Atrophy Medicine Market Definition
1.2 Market Segments
1.2.1 Market by Type
1.2.2 Market by Application
1.3 Global Spinal Muscular Atrophy Medicine 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 Spinal Muscular Atrophy Medicine Overall Market Size
2.1 Global Spinal Muscular Atrophy Medicine Market Size: 2022 VS 2029
2.2 Global Spinal Muscular Atrophy Medicine Revenue, Prospects & Forecasts: 2018-2029
2.3 Global Spinal Muscular Atrophy Medicine Sales: 2018-2029
3 Company Landscape
3.1 Top Spinal Muscular Atrophy Medicine Players in Global Market
3.2 Top Global Spinal Muscular Atrophy Medicine Companies Ranked by Revenue
3.3 Global Spinal Muscular Atrophy Medicine Revenue by Companies
3.4 Global Spinal Muscular Atrophy Medicine Sales by Companies
3.5 Global Spinal Muscular Atrophy Medicine Price by Manufacturer (2018-2023)
3.6 Top 3 and Top 5 Spinal Muscular Atrophy Medicine Companies in Global Market, by Revenue in 2022
3.7 Global Manufacturers Spinal Muscular Atrophy Medicine Product Type
3.8 Tier 1, Tier 2 and Tier 3 Spinal Muscular Atrophy Medicine Players in Global Market
3.8.1 List of Global Tier 1 Spinal Muscular Atrophy Medicine Companies
3.8.2 List of Global Tier 2 and Tier 3 Spinal Muscular Atrophy Medicine Companies
4 Sights by Product
4.1 Overview
4.1.1 By Type – Global Spinal Muscular Atrophy Medicine Market Size Markets, 2022 & 2029
4.1.2 LMI-070
4.1.3 ND-602
4.1.4 NT-1654
4.1.5 Nusinersen
4.1.6 NXD-30001
4.1.7 Others
4.2 By Type – Global Spinal Muscular Atrophy Medicine Revenue & Forecasts
4.2.1 By Type – Global Spinal Muscular Atrophy Medicine Revenue, 2018-2023
4.2.2 By Type – Global Spinal Muscular Atrophy Medicine Revenue, 2024-2029
4.2.3 By Type – Global Spinal Muscular Atrophy Medicine Revenue Market Share, 2018-2029
4.3 By Type – Global Spinal Muscular Atrophy Medicine Sales & Forecasts
4.3.1 By Type – Global Spinal Muscular Atrophy Medicine Sales, 2018-2023
4.3.2 By Type – Global Spinal Muscular Atrophy Medicine Sales, 2024-2029
4.3.3 By Type – Global Spinal Muscular Atrophy Medicine Sales Market Share, 2018-2029
4.4 By Type – Global Spinal Muscular Atrophy Medicine Price (Manufacturers Selling Prices), 2018-2029
5 Sights by Application
5.1 Overview
5.1.1 By Application – Global Spinal Muscular Atrophy Medicine Market Size, 2022 & 2029
5.1.2 Hospital
5.1.3 Clinic
5.1.4 Others
5.2 By Application – Global Spinal Muscular Atrophy Medicine Revenue & Forecasts
5.2.1 By Application – Global Spinal Muscular Atrophy Medicine Revenue, 2018-2023
5.2.2 By Application – Global Spinal Muscular Atrophy Medicine Revenue, 2024-2029
5.2.3 By Application – Global Spinal Muscular Atrophy Medicine Revenue Market Share, 2018-2029
5.3 By Application – Global Spinal Muscular Atrophy Medicine Sales & Forecasts
5.3.1 By Application – Global Spinal Muscular Atrophy Medicine Sales, 2018-2023
5.3.2 By Application – Global Spinal Muscular Atrophy Medicine Sales, 2024-2029
5.3.3 By Application – Global Spinal Muscular Atrophy Medicine Sales Market Share, 2018-2029
5.4 By Application – Global Spinal Muscular Atrophy Medicine Price (Manufacturers Selling Prices), 2018-2029
6 Sights by Region
6.1 By Region – Global Spinal Muscular Atrophy Medicine Market Size, 2022 & 2029
6.2 By Region – Global Spinal Muscular Atrophy Medicine Revenue & Forecasts
6.2.1 By Region – Global Spinal Muscular Atrophy Medicine Revenue, 2018-2023
6.2.2 By Region – Global Spinal Muscular Atrophy Medicine Revenue, 2024-2029
6.2.3 By Region – Global Spinal Muscular Atrophy Medicine Revenue Market Share, 2018-2029
6.3 By Region – Global Spinal Muscular Atrophy Medicine Sales & Forecasts
6.3.1 By Region – Global Spinal Muscular Atrophy Medicine Sales, 2018-2023
6.3.2 By Region – Global Spinal Muscular Atrophy Medicine Sales, 2024-2029
6.3.3 By Region – Global Spinal Muscular Atrophy Medicine Sales Market Share, 2018-2029
6.4 North America
6.4.1 By Country – North America Spinal Muscular Atrophy Medicine Revenue, 2018-2029
6.4.2 By Country – North America Spinal Muscular Atrophy Medicine Sales, 2018-2029
6.4.3 US Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.4.4 Canada Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.4.5 Mexico Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.5 Europe
6.5.1 By Country – Europe Spinal Muscular Atrophy Medicine Revenue, 2018-2029
6.5.2 By Country – Europe Spinal Muscular Atrophy Medicine Sales, 2018-2029
6.5.3 Germany Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.5.4 France Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.5.5 U.K. Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.5.6 Italy Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.5.7 Russia Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.5.8 Nordic Countries Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.5.9 Benelux Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.6 Asia
6.6.1 By Region – Asia Spinal Muscular Atrophy Medicine Revenue, 2018-2029
6.6.2 By Region – Asia Spinal Muscular Atrophy Medicine Sales, 2018-2029
6.6.3 China Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.6.4 Japan Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.6.5 South Korea Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.6.6 Southeast Asia Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.6.7 India Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.7 South America
6.7.1 By Country – South America Spinal Muscular Atrophy Medicine Revenue, 2018-2029
6.7.2 By Country – South America Spinal Muscular Atrophy Medicine Sales, 2018-2029
6.7.3 Brazil Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.7.4 Argentina Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.8 Middle East & Africa
6.8.1 By Country – Middle East & Africa Spinal Muscular Atrophy Medicine Revenue, 2018-2029
6.8.2 By Country – Middle East & Africa Spinal Muscular Atrophy Medicine Sales, 2018-2029
6.8.3 Turkey Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.8.4 Israel Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.8.5 Saudi Arabia Spinal Muscular Atrophy Medicine Market Size, 2018-2029
6.8.6 UAE Spinal Muscular Atrophy Medicine Market Size, 2018-2029
7 Manufacturers & Brands Profiles
7.1 Astellas Pharma Inc.
7.1.1 Astellas Pharma Inc. Company Summary
7.1.2 Astellas Pharma Inc. Business Overview
7.1.3 Astellas Pharma Inc. Spinal Muscular Atrophy Medicine Major Product Offerings
7.1.4 Astellas Pharma Inc. Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.1.5 Astellas Pharma Inc. Key News & Latest Developments
7.2 AveXis, Inc.
7.2.1 AveXis, Inc. Company Summary
7.2.2 AveXis, Inc. Business Overview
7.2.3 AveXis, Inc. Spinal Muscular Atrophy Medicine Major Product Offerings
7.2.4 AveXis, Inc. Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.2.5 AveXis, Inc. Key News & Latest Developments
7.3 Bioblast Pharma Ltd.
7.3.1 Bioblast Pharma Ltd. Company Summary
7.3.2 Bioblast Pharma Ltd. Business Overview
7.3.3 Bioblast Pharma Ltd. Spinal Muscular Atrophy Medicine Major Product Offerings
7.3.4 Bioblast Pharma Ltd. Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.3.5 Bioblast Pharma Ltd. Key News & Latest Developments
7.4 Cytokinetics, Inc. 24
7.4.1 Cytokinetics, Inc. 24 Company Summary
7.4.2 Cytokinetics, Inc. 24 Business Overview
7.4.3 Cytokinetics, Inc. 24 Spinal Muscular Atrophy Medicine Major Product Offerings
7.4.4 Cytokinetics, Inc. 24 Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.4.5 Cytokinetics, Inc. 24 Key News & Latest Developments
7.5 F. Hoffmann-La Roche Ltd.
7.5.1 F. Hoffmann-La Roche Ltd. Company Summary
7.5.2 F. Hoffmann-La Roche Ltd. Business Overview
7.5.3 F. Hoffmann-La Roche Ltd. Spinal Muscular Atrophy Medicine Major Product Offerings
7.5.4 F. Hoffmann-La Roche Ltd. Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.5.5 F. Hoffmann-La Roche Ltd. Key News & Latest Developments
7.6 Genethon
7.6.1 Genethon Company Summary
7.6.2 Genethon Business Overview
7.6.3 Genethon Spinal Muscular Atrophy Medicine Major Product Offerings
7.6.4 Genethon Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.6.5 Genethon Key News & Latest Developments
7.7 Genzyme Corporation
7.7.1 Genzyme Corporation Company Summary
7.7.2 Genzyme Corporation Business Overview
7.7.3 Genzyme Corporation Spinal Muscular Atrophy Medicine Major Product Offerings
7.7.4 Genzyme Corporation Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.7.5 Genzyme Corporation Key News & Latest Developments
7.8 GMP-Orphan SAS
7.8.1 GMP-Orphan SAS Company Summary
7.8.2 GMP-Orphan SAS Business Overview
7.8.3 GMP-Orphan SAS Spinal Muscular Atrophy Medicine Major Product Offerings
7.8.4 GMP-Orphan SAS Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.8.5 GMP-Orphan SAS Key News & Latest Developments
7.9 Ionis Pharmaceuticals, Inc.
7.9.1 Ionis Pharmaceuticals, Inc. Company Summary
7.9.2 Ionis Pharmaceuticals, Inc. Business Overview
7.9.3 Ionis Pharmaceuticals, Inc. Spinal Muscular Atrophy Medicine Major Product Offerings
7.9.4 Ionis Pharmaceuticals, Inc. Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.9.5 Ionis Pharmaceuticals, Inc. Key News & Latest Developments
7.10 Longevity Biotech, Inc
7.10.1 Longevity Biotech, Inc Company Summary
7.10.2 Longevity Biotech, Inc Business Overview
7.10.3 Longevity Biotech, Inc Spinal Muscular Atrophy Medicine Major Product Offerings
7.10.4 Longevity Biotech, Inc Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.10.5 Longevity Biotech, Inc Key News & Latest Developments
7.11 Neurodyn Inc.
7.11.1 Neurodyn Inc. Company Summary
7.11.2 Neurodyn Inc. Spinal Muscular Atrophy Medicine Business Overview
7.11.3 Neurodyn Inc. Spinal Muscular Atrophy Medicine Major Product Offerings
7.11.4 Neurodyn Inc. Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.11.5 Neurodyn Inc. Key News & Latest Developments
7.12 Neurotune AG
7.12.1 Neurotune AG Company Summary
7.12.2 Neurotune AG Spinal Muscular Atrophy Medicine Business Overview
7.12.3 Neurotune AG Spinal Muscular Atrophy Medicine Major Product Offerings
7.12.4 Neurotune AG Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.12.5 Neurotune AG Key News & Latest Developments
7.13 Novartis AG
7.13.1 Novartis AG Company Summary
7.13.2 Novartis AG Spinal Muscular Atrophy Medicine Business Overview
7.13.3 Novartis AG Spinal Muscular Atrophy Medicine Major Product Offerings
7.13.4 Novartis AG Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.13.5 Novartis AG Key News & Latest Developments
7.14 Sarepta Therapeutics, Inc.
7.14.1 Sarepta Therapeutics, Inc. Company Summary
7.14.2 Sarepta Therapeutics, Inc. Business Overview
7.14.3 Sarepta Therapeutics, Inc. Spinal Muscular Atrophy Medicine Major Product Offerings
7.14.4 Sarepta Therapeutics, Inc. Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.14.5 Sarepta Therapeutics, Inc. Key News & Latest Developments
7.15 Voyager Therapeutics, Inc.
7.15.1 Voyager Therapeutics, Inc. Company Summary
7.15.2 Voyager Therapeutics, Inc. Business Overview
7.15.3 Voyager Therapeutics, Inc. Spinal Muscular Atrophy Medicine Major Product Offerings
7.15.4 Voyager Therapeutics, Inc. Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.15.5 Voyager Therapeutics, Inc. Key News & Latest Developments
7.16 Vybion, Inc.
7.16.1 Vybion, Inc. Company Summary
7.16.2 Vybion, Inc. Business Overview
7.16.3 Vybion, Inc. Spinal Muscular Atrophy Medicine Major Product Offerings
7.16.4 Vybion, Inc. Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.16.5 Vybion, Inc. Key News & Latest Developments
7.17 WAVE Life Sciences Ltd.
7.17.1 WAVE Life Sciences Ltd. Company Summary
7.17.2 WAVE Life Sciences Ltd. Business Overview
7.17.3 WAVE Life Sciences Ltd. Spinal Muscular Atrophy Medicine Major Product Offerings
7.17.4 WAVE Life Sciences Ltd. Spinal Muscular Atrophy Medicine Sales and Revenue in Global (2018-2023)
7.17.5 WAVE Life Sciences Ltd. Key News & Latest Developments
8 Global Spinal Muscular Atrophy Medicine Production Capacity, Analysis
8.1 Global Spinal Muscular Atrophy Medicine Production Capacity, 2018-2029
8.2 Spinal Muscular Atrophy Medicine Production Capacity of Key Manufacturers in Global Market
8.3 Global Spinal Muscular Atrophy Medicine Production by Region
9 Key Market Trends, Opportunity, Drivers and Restraints
9.1 Market Opportunities & Trends
9.2 Market Drivers
9.3 Market Restraints
10 Spinal Muscular Atrophy Medicine Supply Chain Analysis
10.1 Spinal Muscular Atrophy Medicine Industry Value Chain
10.2 Spinal Muscular Atrophy Medicine Upstream Market
10.3 Spinal Muscular Atrophy Medicine Downstream and Clients
10.4 Marketing Channels Analysis
10.4.1 Marketing Channels
10.4.2 Spinal Muscular Atrophy Medicine Distributors and Sales Agents in Global
11 Conclusion
12 Appendix
12.1 Note
12.2 Examples of Clients
12.3 Disclaimer
Table 1. Key Players of Spinal Muscular Atrophy Medicine in Global Market
Table 2. Top Spinal Muscular Atrophy Medicine Players in Global Market, Ranking by Revenue (2022)
Table 3. Global Spinal Muscular Atrophy Medicine Revenue by Companies, (US$, Mn), 2018-2023
Table 4. Global Spinal Muscular Atrophy Medicine Revenue Share by Companies, 2018-2023
Table 5. Global Spinal Muscular Atrophy Medicine Sales by Companies, (K Pcs), 2018-2023
Table 6. Global Spinal Muscular Atrophy Medicine Sales Share by Companies, 2018-2023
Table 7. Key Manufacturers Spinal Muscular Atrophy Medicine Price (2018-2023) & (USD/Pcs)
Table 8. Global Manufacturers Spinal Muscular Atrophy Medicine Product Type
Table 9. List of Global Tier 1 Spinal Muscular Atrophy Medicine Companies, Revenue (US$, Mn) in 2022 and Market Share
Table 10. List of Global Tier 2 and Tier 3 Spinal Muscular Atrophy Medicine Companies, Revenue (US$, Mn) in 2022 and Market Share
Table 11. By Type – Global Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2022 & 2029
Table 12. By Type - Global Spinal Muscular Atrophy Medicine Revenue (US$, Mn), 2018-2023
Table 13. By Type - Global Spinal Muscular Atrophy Medicine Revenue (US$, Mn), 2024-2029
Table 14. By Type - Global Spinal Muscular Atrophy Medicine Sales (K Pcs), 2018-2023
Table 15. By Type - Global Spinal Muscular Atrophy Medicine Sales (K Pcs), 2024-2029
Table 16. By Application – Global Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2022 & 2029
Table 17. By Application - Global Spinal Muscular Atrophy Medicine Revenue (US$, Mn), 2018-2023
Table 18. By Application - Global Spinal Muscular Atrophy Medicine Revenue (US$, Mn), 2024-2029
Table 19. By Application - Global Spinal Muscular Atrophy Medicine Sales (K Pcs), 2018-2023
Table 20. By Application - Global Spinal Muscular Atrophy Medicine Sales (K Pcs), 2024-2029
Table 21. By Region – Global Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2022 VS 2029
Table 22. By Region - Global Spinal Muscular Atrophy Medicine Revenue (US$, Mn), 2018-2023
Table 23. By Region - Global Spinal Muscular Atrophy Medicine Revenue (US$, Mn), 2024-2029
Table 24. By Region - Global Spinal Muscular Atrophy Medicine Sales (K Pcs), 2018-2023
Table 25. By Region - Global Spinal Muscular Atrophy Medicine Sales (K Pcs), 2024-2029
Table 26. By Country - North America Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2023
Table 27. By Country - North America Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2024-2029
Table 28. By Country - North America Spinal Muscular Atrophy Medicine Sales, (K Pcs), 2018-2023
Table 29. By Country - North America Spinal Muscular Atrophy Medicine Sales, (K Pcs), 2024-2029
Table 30. By Country - Europe Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2023
Table 31. By Country - Europe Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2024-2029
Table 32. By Country - Europe Spinal Muscular Atrophy Medicine Sales, (K Pcs), 2018-2023
Table 33. By Country - Europe Spinal Muscular Atrophy Medicine Sales, (K Pcs), 2024-2029
Table 34. By Region - Asia Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2023
Table 35. By Region - Asia Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2024-2029
Table 36. By Region - Asia Spinal Muscular Atrophy Medicine Sales, (K Pcs), 2018-2023
Table 37. By Region - Asia Spinal Muscular Atrophy Medicine Sales, (K Pcs), 2024-2029
Table 38. By Country - South America Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2023
Table 39. By Country - South America Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2024-2029
Table 40. By Country - South America Spinal Muscular Atrophy Medicine Sales, (K Pcs), 2018-2023
Table 41. By Country - South America Spinal Muscular Atrophy Medicine Sales, (K Pcs), 2024-2029
Table 42. By Country - Middle East & Africa Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2023
Table 43. By Country - Middle East & Africa Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2024-2029
Table 44. By Country - Middle East & Africa Spinal Muscular Atrophy Medicine Sales, (K Pcs), 2018-2023
Table 45. By Country - Middle East & Africa Spinal Muscular Atrophy Medicine Sales, (K Pcs), 2024-2029
Table 46. Astellas Pharma Inc. Company Summary
Table 47. Astellas Pharma Inc. Spinal Muscular Atrophy Medicine Product Offerings
Table 48. Astellas Pharma Inc. Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 49. Astellas Pharma Inc. Key News & Latest Developments
Table 50. AveXis, Inc. Company Summary
Table 51. AveXis, Inc. Spinal Muscular Atrophy Medicine Product Offerings
Table 52. AveXis, Inc. Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 53. AveXis, Inc. Key News & Latest Developments
Table 54. Bioblast Pharma Ltd. Company Summary
Table 55. Bioblast Pharma Ltd. Spinal Muscular Atrophy Medicine Product Offerings
Table 56. Bioblast Pharma Ltd. Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 57. Bioblast Pharma Ltd. Key News & Latest Developments
Table 58. Cytokinetics, Inc. 24 Company Summary
Table 59. Cytokinetics, Inc. 24 Spinal Muscular Atrophy Medicine Product Offerings
Table 60. Cytokinetics, Inc. 24 Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 61. Cytokinetics, Inc. 24 Key News & Latest Developments
Table 62. F. Hoffmann-La Roche Ltd. Company Summary
Table 63. F. Hoffmann-La Roche Ltd. Spinal Muscular Atrophy Medicine Product Offerings
Table 64. F. Hoffmann-La Roche Ltd. Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 65. F. Hoffmann-La Roche Ltd. Key News & Latest Developments
Table 66. Genethon Company Summary
Table 67. Genethon Spinal Muscular Atrophy Medicine Product Offerings
Table 68. Genethon Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 69. Genethon Key News & Latest Developments
Table 70. Genzyme Corporation Company Summary
Table 71. Genzyme Corporation Spinal Muscular Atrophy Medicine Product Offerings
Table 72. Genzyme Corporation Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 73. Genzyme Corporation Key News & Latest Developments
Table 74. GMP-Orphan SAS Company Summary
Table 75. GMP-Orphan SAS Spinal Muscular Atrophy Medicine Product Offerings
Table 76. GMP-Orphan SAS Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 77. GMP-Orphan SAS Key News & Latest Developments
Table 78. Ionis Pharmaceuticals, Inc. Company Summary
Table 79. Ionis Pharmaceuticals, Inc. Spinal Muscular Atrophy Medicine Product Offerings
Table 80. Ionis Pharmaceuticals, Inc. Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 81. Ionis Pharmaceuticals, Inc. Key News & Latest Developments
Table 82. Longevity Biotech, Inc Company Summary
Table 83. Longevity Biotech, Inc Spinal Muscular Atrophy Medicine Product Offerings
Table 84. Longevity Biotech, Inc Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 85. Longevity Biotech, Inc Key News & Latest Developments
Table 86. Neurodyn Inc. Company Summary
Table 87. Neurodyn Inc. Spinal Muscular Atrophy Medicine Product Offerings
Table 88. Neurodyn Inc. Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 89. Neurodyn Inc. Key News & Latest Developments
Table 90. Neurotune AG Company Summary
Table 91. Neurotune AG Spinal Muscular Atrophy Medicine Product Offerings
Table 92. Neurotune AG Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 93. Neurotune AG Key News & Latest Developments
Table 94. Novartis AG Company Summary
Table 95. Novartis AG Spinal Muscular Atrophy Medicine Product Offerings
Table 96. Novartis AG Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 97. Novartis AG Key News & Latest Developments
Table 98. Sarepta Therapeutics, Inc. Company Summary
Table 99. Sarepta Therapeutics, Inc. Spinal Muscular Atrophy Medicine Product Offerings
Table 100. Sarepta Therapeutics, Inc. Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 101. Sarepta Therapeutics, Inc. Key News & Latest Developments
Table 102. Voyager Therapeutics, Inc. Company Summary
Table 103. Voyager Therapeutics, Inc. Spinal Muscular Atrophy Medicine Product Offerings
Table 104. Voyager Therapeutics, Inc. Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 105. Voyager Therapeutics, Inc. Key News & Latest Developments
Table 106. Vybion, Inc. Company Summary
Table 107. Vybion, Inc. Spinal Muscular Atrophy Medicine Product Offerings
Table 108. Vybion, Inc. Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 109. Vybion, Inc. Key News & Latest Developments
Table 110. WAVE Life Sciences Ltd. Company Summary
Table 111. WAVE Life Sciences Ltd. Spinal Muscular Atrophy Medicine Product Offerings
Table 112. WAVE Life Sciences Ltd. Spinal Muscular Atrophy Medicine Sales (K Pcs), Revenue (US$, Mn) and Average Price (USD/Pcs) (2018-2023)
Table 113. WAVE Life Sciences Ltd. Key News & Latest Developments
Table 114. Spinal Muscular Atrophy Medicine Production Capacity (K Pcs) of Key Manufacturers in Global Market, 2021-2023 (K Pcs)
Table 115. Global Spinal Muscular Atrophy Medicine Capacity Market Share of Key Manufacturers, 2021-2023
Table 116. Global Spinal Muscular Atrophy Medicine Production by Region, 2018-2023 (K Pcs)
Table 117. Global Spinal Muscular Atrophy Medicine Production by Region, 2024-2029 (K Pcs)
Table 118. Spinal Muscular Atrophy Medicine Market Opportunities & Trends in Global Market
Table 119. Spinal Muscular Atrophy Medicine Market Drivers in Global Market
Table 120. Spinal Muscular Atrophy Medicine Market Restraints in Global Market
Table 121. Spinal Muscular Atrophy Medicine Raw Materials
Table 122. Spinal Muscular Atrophy Medicine Raw Materials Suppliers in Global Market
Table 123. Typical Spinal Muscular Atrophy Medicine Downstream
Table 124. Spinal Muscular Atrophy Medicine Downstream Clients in Global Market
Table 125. Spinal Muscular Atrophy Medicine Distributors and Sales Agents in Global Market
List of Figures
Figure 1. Spinal Muscular Atrophy Medicine Segment by Type in 2022
Figure 2. Spinal Muscular Atrophy Medicine Segment by Application in 2022
Figure 3. Global Spinal Muscular Atrophy Medicine Market Overview: 2022
Figure 4. Key Caveats
Figure 5. Global Spinal Muscular Atrophy Medicine Market Size: 2022 VS 2029 (US$, Mn)
Figure 6. Global Spinal Muscular Atrophy Medicine Revenue, 2018-2029 (US$, Mn)
Figure 7. Spinal Muscular Atrophy Medicine Sales in Global Market: 2018-2029 (K Pcs)
Figure 8. The Top 3 and 5 Players Market Share by Spinal Muscular Atrophy Medicine Revenue in 2022
Figure 9. By Type - Global Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2022 & 2029
Figure 10. By Type - Global Spinal Muscular Atrophy Medicine Revenue Market Share, 2018-2029
Figure 11. By Type - Global Spinal Muscular Atrophy Medicine Sales Market Share, 2018-2029
Figure 12. By Type - Global Spinal Muscular Atrophy Medicine Price (USD/Pcs), 2018-2029
Figure 13. By Application - Global Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2022 & 2029
Figure 14. By Application - Global Spinal Muscular Atrophy Medicine Revenue Market Share, 2018-2029
Figure 15. By Application - Global Spinal Muscular Atrophy Medicine Sales Market Share, 2018-2029
Figure 16. By Application - Global Spinal Muscular Atrophy Medicine Price (USD/Pcs), 2018-2029
Figure 17. By Region - Global Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2022 & 2029
Figure 18. By Region - Global Spinal Muscular Atrophy Medicine Revenue Market Share, 2018 VS 2022 VS 2029
Figure 19. By Region - Global Spinal Muscular Atrophy Medicine Revenue Market Share, 2018-2029
Figure 20. By Region - Global Spinal Muscular Atrophy Medicine Sales Market Share, 2018-2029
Figure 21. By Country - North America Spinal Muscular Atrophy Medicine Revenue Market Share, 2018-2029
Figure 22. By Country - North America Spinal Muscular Atrophy Medicine Sales Market Share, 2018-2029
Figure 23. US Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 24. Canada Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 25. Mexico Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 26. By Country - Europe Spinal Muscular Atrophy Medicine Revenue Market Share, 2018-2029
Figure 27. By Country - Europe Spinal Muscular Atrophy Medicine Sales Market Share, 2018-2029
Figure 28. Germany Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 29. France Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 30. U.K. Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 31. Italy Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 32. Russia Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 33. Nordic Countries Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 34. Benelux Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 35. By Region - Asia Spinal Muscular Atrophy Medicine Revenue Market Share, 2018-2029
Figure 36. By Region - Asia Spinal Muscular Atrophy Medicine Sales Market Share, 2018-2029
Figure 37. China Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 38. Japan Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 39. South Korea Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 40. Southeast Asia Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 41. India Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 42. By Country - South America Spinal Muscular Atrophy Medicine Revenue Market Share, 2018-2029
Figure 43. By Country - South America Spinal Muscular Atrophy Medicine Sales Market Share, 2018-2029
Figure 44. Brazil Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 45. Argentina Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 46. By Country - Middle East & Africa Spinal Muscular Atrophy Medicine Revenue Market Share, 2018-2029
Figure 47. By Country - Middle East & Africa Spinal Muscular Atrophy Medicine Sales Market Share, 2018-2029
Figure 48. Turkey Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 49. Israel Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 50. Saudi Arabia Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 51. UAE Spinal Muscular Atrophy Medicine Revenue, (US$, Mn), 2018-2029
Figure 52. Global Spinal Muscular Atrophy Medicine Production Capacity (K Pcs), 2018-2029
Figure 53. The Percentage of Production Spinal Muscular Atrophy Medicine by Region, 2022 VS 2029
Figure 54. Spinal Muscular Atrophy Medicine Industry Value Chain
Figure 55. Marketing Channels
※参考情報 脊髄性筋萎縮症(Spinal Muscular Atrophy、SMA)は、運動ニューロンが障害されることにより引き起こされる遺伝性の神経筋疾患です。主に脊髄の前角細胞が影響を受け、筋力の低下や筋萎縮を引き起こします。この疾患は遺伝に基づいており、常染色体劣性遺伝形式で遺伝することが一般的です。脊髄性筋萎縮症は、主にSMA1(上位型)、SMA2(中間型)、SMA3(下位型)、SMA4(成人型)の4つの主要なタイプに分類されます。 SMA1は最も重篤なタイプで、生後6ヶ月以内に症状が現れ、通常は2歳になる前に生命を脅かす合併症が発生します。SMA2は生後6ヶ月から18ヶ月の間に症状が現れ、座ることはできるが、歩行能力はないことが特徴です。SMA3は4歳以降に発症し、通常は歩行能力があり、時に症状が進行することで筋力が低下していきます。SMA4は成人型で、通常は30歳以降に発症し、筋力低下が緩やかで、比較的良好な生活が可能な場合が多いです。 脊髄性筋萎縮症の発症は、SMN遺伝子(Survival Motor Neuron Gene)に関連しています。通常、SMN1遺伝子が正常に機能することで運動ニューロンが生存し、健康な筋肉を維持します。しかし、SMAの患者では、SMN1遺伝子の欠失や変異が見られ、この遺伝子の機能が低下することで、運動ニューロンが徐々に死んでしまいます。その結果、筋肉が萎縮し、様々な運動能力に影響を与えます。 治療に関しては、近年、脊髄性筋萎縮症へのアプローチが劇的に進化を遂げてきました。従来は対症療法が中心でしたが、現在では病因に基づく治療法が開発されています。具体的には、遺伝子治療薬である「スピンラザ(Nusinersen)」や、遺伝子編集技術を利用した「Zolgensma(Onasemnogene abeparvovec)」が注目されています。スピンラザは、SMN2遺伝子のスプライシングを改善し、正常なSMNタンパク質の産生を促すことによって運動ニューロンの機能を向上させます。一方、Zolgensmaは、欠損しているSMN1遺伝子を補うために、ウイルスベクターを使用して正常なSMN1遺伝子を体内に導入します。これにより、運動ニューロンが生存しやすくなり、筋力の維持となることが期待されています。 さらに、最近ではリハビリテーションや他の支持療法も含めた包括的な治療が重要視されています。理学療法や作業療法を通じて、筋力維持や日常生活の質の向上を図ることが、それぞれのタイプに適した形式で行われています。このような多面的なアプローチにより、患者はより良い生活を送ることが可能になっています。 脊髄性筋萎縮症の研究は、これまで以上に活発になっており、今後新たな治療法や治験が期待されています。特に、遺伝子治療の分野では、さらなる改善や副作用軽減が求められています。また、患者の早期診断を可能にするためのバイオマーカーの発見も重要です。これにより、より早期に介入し、後遺症を最小限に抑えることができる可能性があります。 加えて、SMA患者やその家族に対するサポート体制の充実も求められています。患者団体や支援団体が積極的に活動しており、情報提供や支援を行っています。医療者は、患者や家族に対して最新の情報を提供するだけでなく、精神的なサポートやコミュニティの構築も重要です。これにより、患者自身が自分の状況を理解し、前向きに治療に取り組むための基盤を整えることができます。 脊髄性筋萎縮症の治療は、個々の患者の状況に応じて柔軟にアプローチすることが必要です。これにより、患者の生命予後や生活の質を向上させ、より良い治療結果を実現することが期待されます。治療の進歩とともに、SMA患者の未来には明るい希望が広がっています。研究者や医療従事者、患者やその家族が協力し合いながら、脊髄性筋萎縮症の理解と治療の向上を目指していくことが重要です。世界中の研究者たちの努力が、将来的には全てのSMA患者に恩恵をもたらすことを期待しています。脊髄性筋萎縮症は、依然として課題の多い疾患ですが、患者とその家族にとって明るい未来を実現するための努力が続けられています。 |
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