Charcot-Marie-Tooth Disease Global Market Report 2025

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.