The charcot-marie-tooth disease market size has grown exponentially in recent years. It will grow from $1.09 billion in 2024 to $1.32 billion in 2025 at a compound annual growth rate (CAGR) of 20.9%. Growth during the historic period can be attributed to rising public awareness of genetic neuropathies, increased utilization of genetic counseling services, greater physician education on rare diseases, strengthened patient advocacy initiatives, and broader access to neurophysiological testing.
The charcot-marie-tooth disease market size is expected to see exponential growth in the next few years. It will grow to $2.8 billion in 2029 at a compound annual growth rate (CAGR) of 20.6%. Growth in the forecast period can be attributed to the implementation of early screening programs for inherited disorders, the expansion of neurology and neuromuscular specialty clinics, improved reimbursement policies for rare disease treatments, broader insurance coverage for genetic testing, and increased healthcare spending. Key trends expected during this period include ongoing clinical trials for disease-modifying therapies, a strong pipeline of promising drug candidates, growing investment by pharmaceutical companies in rare disease treatments, orphan drug designations that enhance research and development incentives, and advancements in gene therapy and gene editing technologies.
The rise in healthcare expenditure is expected to drive the growth of the Charcot-Marie-Tooth (CMT) disease market in the coming years. Healthcare expenditure refers to the total spending on medical services, supplies, and activities focused on maintaining or improving health. This expenditure is increasing due to the advancement and adoption of cutting-edge medical technologies, which require significant investment in research, equipment, and specialized training - contributing to higher overall healthcare costs. The growth in healthcare spending facilitates greater investment in CMT treatment by enhancing access to specialized therapies, diagnostic tools, and supportive care services, leading to improved disease management. For example, in April 2025, the Office for National Statistics, a UK-based government agency, reported a 6.5% increase in healthcare spending from 2023 to 2024, slightly higher than the 6.3% rise recorded in 2023. Thus, the continued surge in healthcare expenditure is set to support the expansion of the Charcot-Marie-Tooth disease market.
Leading companies in the Charcot-Marie-Tooth (CMT) disease market are concentrating on developing advanced therapies, such as mesenchymal stem cell treatments, which offer ease of isolation and expansion from various tissue sources. Mesenchymal stem cell therapies utilize versatile cells capable of differentiating into multiple tissue types to promote healing and reduce inflammation in diverse health conditions. For example, in March 2025, ENCell Co. Ltd., a South Korea-based biopharmaceutical firm, announced that the U.S. Food and Drug Administration granted orphan drug designation to EN001, a mesenchymal stem cell therapy designed to treat CMT disease. EN001 aims to enhance cell survival and boost the release of therapeutic molecules. Upon administration, the therapy targets nerve repair, stimulates the release of healing factors, and supports nerve remyelination. A phase 1 open-label, dose-escalation clinical trial (NCT05333406) was conducted involving patients with type 1a CMT to evaluate safety, tolerability, and optimal dosing over 16 weeks, following a 3+3 trial design. Participants in the low-dose group received an intravenous dose on the first day.
In November 2024, the Charcot-Marie-Tooth Association (CMTA), a U.S.-based non-profit organization, partnered with the Inherited Neuropathy Consortium (INC) to advance comprehensive research across all variants of Charcot-Marie-Tooth disease. This collaboration aims to accelerate the translation of research into clinical applications, pushing treatments forward and offering hope to individuals affected by Charcot-Marie-Tooth. The Inherited Neuropathy Consortium (INC) is a U.S.-based clinical research consortium focused on rare diseases.
Major players in the charcot-marie-tooth disease market are F-Hoffmann-La-Roche-Ltd, Sanofi SA, Bristol-Myers Squibb Company, Novartis AG, Biogen Inc., Sarepta Therapeutics Inc., Chong Kun Dang Pharmaceutical Corp., Ionis Pharmaceuticals Inc., Ultragenyx Pharmaceutical Inc., Acceleron Pharma, Affectis Pharmaceuticals AG, NMD Pharma, Pharnext SA, MedDay Pharmaceuticals, Inflectis BioScience, Helixmith Co. Ltd., Addex Therapeutics, Augustine Therapeutics, DTx Pharma Inc., ENCell Co.
North America was the largest region in the charcot-marie-tooth disease market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in charcot-marie-tooth disease report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.
The countries covered in the charcot-marie-tooth disease market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
Charcot-marie-tooth (CMT) disease refers to a group of inherited neurological disorders that impact the peripheral nerves, which are responsible for muscle control and transmitting sensory information from the limbs to the brain. It is marked by progressive muscle weakness and atrophy, particularly in the feet, lower legs, hands, and forearms, often accompanied by sensory loss, foot deformities (such as high arches or hammertoes), and difficulty with walking. CMT is caused by genetic mutations that disrupt the structure or function of peripheral nerves. The condition usually begins during adolescence or early adulthood, though the age of onset and severity may differ among individuals.
The primary types of charcot-marie-tooth disease include CMT 1, CMT 2, CMT 3, and others. Type 1 CMT (CMT 1) is a hereditary neuropathy resulting from abnormalities in the myelin sheath that insulates peripheral nerves. Treatment involves various drug types, including nonsteroidal anti-inflammatory drugs (NSAIDs), cyclooxygenase-2 inhibitors, tricyclic antidepressants, anticonvulsants, analgesics, and drugs currently in the pipeline. These medications are distributed through hospital pharmacies, retail pharmacies, and online pharmacies.
The charcot-marie-tooth disease market research report is one of a series of new reports that provides charcot-marie-tooth disease market statistics, including charcot-marie-tooth disease industry global market size, regional shares, competitors with a charcot-marie-tooth disease market share, detailed charcot-marie-tooth disease market segments, market trends and opportunities, and any further data you may need to thrive in the charcot-marie-tooth disease industry. This charcot-marie-tooth disease market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.
The charcot-marie-tooth disease market consists of revenues earned by entities by providing services such as genetic testing services, physical therapy services, occupational therapy services, and orthotic fitting services. The market value includes the value of related goods sold by the service provider or included within the service offering. The charcot-marie-tooth disease market also includes sales of custom insoles, mobility aids, electrostimulation devices, and compression garments. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
The charcot-marie-tooth disease market size is expected to see exponential growth in the next few years. It will grow to $2.8 billion in 2029 at a compound annual growth rate (CAGR) of 20.6%. Growth in the forecast period can be attributed to the implementation of early screening programs for inherited disorders, the expansion of neurology and neuromuscular specialty clinics, improved reimbursement policies for rare disease treatments, broader insurance coverage for genetic testing, and increased healthcare spending. Key trends expected during this period include ongoing clinical trials for disease-modifying therapies, a strong pipeline of promising drug candidates, growing investment by pharmaceutical companies in rare disease treatments, orphan drug designations that enhance research and development incentives, and advancements in gene therapy and gene editing technologies.
The rise in healthcare expenditure is expected to drive the growth of the Charcot-Marie-Tooth (CMT) disease market in the coming years. Healthcare expenditure refers to the total spending on medical services, supplies, and activities focused on maintaining or improving health. This expenditure is increasing due to the advancement and adoption of cutting-edge medical technologies, which require significant investment in research, equipment, and specialized training - contributing to higher overall healthcare costs. The growth in healthcare spending facilitates greater investment in CMT treatment by enhancing access to specialized therapies, diagnostic tools, and supportive care services, leading to improved disease management. For example, in April 2025, the Office for National Statistics, a UK-based government agency, reported a 6.5% increase in healthcare spending from 2023 to 2024, slightly higher than the 6.3% rise recorded in 2023. Thus, the continued surge in healthcare expenditure is set to support the expansion of the Charcot-Marie-Tooth disease market.
Leading companies in the Charcot-Marie-Tooth (CMT) disease market are concentrating on developing advanced therapies, such as mesenchymal stem cell treatments, which offer ease of isolation and expansion from various tissue sources. Mesenchymal stem cell therapies utilize versatile cells capable of differentiating into multiple tissue types to promote healing and reduce inflammation in diverse health conditions. For example, in March 2025, ENCell Co. Ltd., a South Korea-based biopharmaceutical firm, announced that the U.S. Food and Drug Administration granted orphan drug designation to EN001, a mesenchymal stem cell therapy designed to treat CMT disease. EN001 aims to enhance cell survival and boost the release of therapeutic molecules. Upon administration, the therapy targets nerve repair, stimulates the release of healing factors, and supports nerve remyelination. A phase 1 open-label, dose-escalation clinical trial (NCT05333406) was conducted involving patients with type 1a CMT to evaluate safety, tolerability, and optimal dosing over 16 weeks, following a 3+3 trial design. Participants in the low-dose group received an intravenous dose on the first day.
In November 2024, the Charcot-Marie-Tooth Association (CMTA), a U.S.-based non-profit organization, partnered with the Inherited Neuropathy Consortium (INC) to advance comprehensive research across all variants of Charcot-Marie-Tooth disease. This collaboration aims to accelerate the translation of research into clinical applications, pushing treatments forward and offering hope to individuals affected by Charcot-Marie-Tooth. The Inherited Neuropathy Consortium (INC) is a U.S.-based clinical research consortium focused on rare diseases.
Major players in the charcot-marie-tooth disease market are F-Hoffmann-La-Roche-Ltd, Sanofi SA, Bristol-Myers Squibb Company, Novartis AG, Biogen Inc., Sarepta Therapeutics Inc., Chong Kun Dang Pharmaceutical Corp., Ionis Pharmaceuticals Inc., Ultragenyx Pharmaceutical Inc., Acceleron Pharma, Affectis Pharmaceuticals AG, NMD Pharma, Pharnext SA, MedDay Pharmaceuticals, Inflectis BioScience, Helixmith Co. Ltd., Addex Therapeutics, Augustine Therapeutics, DTx Pharma Inc., ENCell Co.
North America was the largest region in the charcot-marie-tooth disease market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in charcot-marie-tooth disease report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.
The countries covered in the charcot-marie-tooth disease market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
Charcot-marie-tooth (CMT) disease refers to a group of inherited neurological disorders that impact the peripheral nerves, which are responsible for muscle control and transmitting sensory information from the limbs to the brain. It is marked by progressive muscle weakness and atrophy, particularly in the feet, lower legs, hands, and forearms, often accompanied by sensory loss, foot deformities (such as high arches or hammertoes), and difficulty with walking. CMT is caused by genetic mutations that disrupt the structure or function of peripheral nerves. The condition usually begins during adolescence or early adulthood, though the age of onset and severity may differ among individuals.
The primary types of charcot-marie-tooth disease include CMT 1, CMT 2, CMT 3, and others. Type 1 CMT (CMT 1) is a hereditary neuropathy resulting from abnormalities in the myelin sheath that insulates peripheral nerves. Treatment involves various drug types, including nonsteroidal anti-inflammatory drugs (NSAIDs), cyclooxygenase-2 inhibitors, tricyclic antidepressants, anticonvulsants, analgesics, and drugs currently in the pipeline. These medications are distributed through hospital pharmacies, retail pharmacies, and online pharmacies.
The charcot-marie-tooth disease market research report is one of a series of new reports that provides charcot-marie-tooth disease market statistics, including charcot-marie-tooth disease industry global market size, regional shares, competitors with a charcot-marie-tooth disease market share, detailed charcot-marie-tooth disease market segments, market trends and opportunities, and any further data you may need to thrive in the charcot-marie-tooth disease industry. This charcot-marie-tooth disease market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.
The charcot-marie-tooth disease market consists of revenues earned by entities by providing services such as genetic testing services, physical therapy services, occupational therapy services, and orthotic fitting services. The market value includes the value of related goods sold by the service provider or included within the service offering. The charcot-marie-tooth disease market also includes sales of custom insoles, mobility aids, electrostimulation devices, and compression garments. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
Table of Contents
1. Executive Summary2. Charcot-Marie-Tooth Disease Market Characteristics3. Charcot-Marie-Tooth Disease Market Trends And Strategies4. Charcot-Marie-Tooth Disease Market - Macro Economic Scenario Including The Impact Of Interest Rates, Inflation, Geopolitics, Trade Wars and Tariffs, And Covid And Recovery On The Market32. Global Charcot-Marie-Tooth Disease Market Competitive Benchmarking And Dashboard33. Key Mergers And Acquisitions In The Charcot-Marie-Tooth Disease Market34. Recent Developments In The Charcot-Marie-Tooth Disease Market
5. Global Charcot-Marie-Tooth Disease Growth Analysis And Strategic Analysis Framework
6. Charcot-Marie-Tooth Disease Market Segmentation
7. Charcot-Marie-Tooth Disease Market Regional And Country Analysis
8. Asia-Pacific Charcot-Marie-Tooth Disease Market
9. China Charcot-Marie-Tooth Disease Market
10. India Charcot-Marie-Tooth Disease Market
11. Japan Charcot-Marie-Tooth Disease Market
12. Australia Charcot-Marie-Tooth Disease Market
13. Indonesia Charcot-Marie-Tooth Disease Market
14. South Korea Charcot-Marie-Tooth Disease Market
15. Western Europe Charcot-Marie-Tooth Disease Market
16. UK Charcot-Marie-Tooth Disease Market
17. Germany Charcot-Marie-Tooth Disease Market
18. France Charcot-Marie-Tooth Disease Market
19. Italy Charcot-Marie-Tooth Disease Market
20. Spain Charcot-Marie-Tooth Disease Market
21. Eastern Europe Charcot-Marie-Tooth Disease Market
22. Russia Charcot-Marie-Tooth Disease Market
23. North America Charcot-Marie-Tooth Disease Market
24. USA Charcot-Marie-Tooth Disease Market
25. Canada Charcot-Marie-Tooth Disease Market
26. South America Charcot-Marie-Tooth Disease Market
27. Brazil Charcot-Marie-Tooth Disease Market
28. Middle East Charcot-Marie-Tooth Disease Market
29. Africa Charcot-Marie-Tooth Disease Market
30. Charcot-Marie-Tooth Disease Market Competitive Landscape And Company Profiles
31. Charcot-Marie-Tooth Disease Market Other Major And Innovative Companies
35. Charcot-Marie-Tooth Disease Market High Potential Countries, Segments and Strategies
36. Appendix
Executive Summary
Charcot-Marie-Tooth Disease Global Market Report 2025 provides strategists, marketers and senior management with the critical information they need to assess the market.This report focuses on charcot-marie-tooth disease market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.
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Description
Where is the largest and fastest growing market for charcot-marie-tooth disease ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The charcot-marie-tooth disease market global report answers all these questions and many more.The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market’s historic and forecast market growth by geography.
- The market characteristics section of the report defines and explains the market.
- The market size section gives the market size ($b) covering both the historic growth of the market, and forecasting its development.
- The forecasts are made after considering the major factors currently impacting the market. These include:
- The forecasts are made after considering the major factors currently impacting the market. These include the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.
- Market segmentations break down the market into sub markets.
- The regional and country breakdowns section gives an analysis of the market in each geography and the size of the market by geography and compares their historic and forecast growth. It covers the growth trajectory of COVID-19 for all regions, key developed countries and major emerging markets.
- The competitive landscape chapter gives a description of the competitive nature of the market, market shares, and a description of the leading companies. Key financial deals which have shaped the market in recent years are identified.
- The trends and strategies section analyses the shape of the market as it emerges from the crisis and suggests how companies can grow as the market recovers.
Scope
Markets Covered:
1) By Disease Type: CMT 1; CMT 2; CMT 3; Other Disease Types2) By Drug Type: Nonsteroidal Anti-Inflammatory Drugs; Cyclooxygenase-2 Inhibitors; Tricyclic Antidepressants; Anticonvulsants; Analgesics; Pipeline Drug
3) By Distribution Channel: Hospital Pharmacies; Retail Pharmacies; Online Pharmacies
Key Companies Profiled: F-Hoffmann-La-Roche-Ltd; Sanofi SA; Bristol-Myers Squibb Company; Novartis AG; Biogen Inc.
Countries: Australia; Brazil; China; France; Germany; India; Indonesia; Japan; Russia; South Korea; UK; USA; Canada; Italy; Spain
Regions: Asia-Pacific; Western Europe; Eastern Europe; North America; South America; Middle East; Africa
Time Series: Five years historic and ten years forecast.
Data: Ratios of market size and growth to related markets, GDP proportions, expenditure per capita.
Data Segmentation: Country and regional historic and forecast data, market share of competitors, market segments.
Sourcing and Referencing: Data and analysis throughout the report is sourced using end notes.
Delivery Format: PDF, Word and Excel Data Dashboard.
Companies Mentioned
- F-Hoffmann-La-Roche-Ltd
- Sanofi SA
- Bristol-Myers Squibb Company
- Novartis AG
- Biogen Inc.
- Sarepta Therapeutics Inc.
- Chong Kun Dang Pharmaceutical Corp.
- Ionis Pharmaceuticals Inc.
- Ultragenyx Pharmaceutical Inc.
- Acceleron Pharma
- Affectis Pharmaceuticals AG
- NMD Pharma
- Pharnext SA
- MedDay Pharmaceuticals
- Inflectis BioScience
- Helixmith Co. Ltd.
- Addex Therapeutics
- Augustine Therapeutics
- DTx Pharma Inc.
- ENCell Co