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United States Charcot-Marie-Tooth Disease Market to Grow at 5.8% CAGR, Led by Neurogene, Pharnext, and Affectis Pharmaceuticals | DataM Intelligence
Charcot-Marie-Tooth Disease Market
United States: Recent Industry Developments
✅ In October 2025, Neurogene Inc. received FDA Orphan Drug Designation for its novel gene therapy targeting CMT1A, moving into Phase III clinical trials.
✅ In October 2025, the National Institutes of Health (NIH) allocated USD 45 million for CMT research focusing on genetic therapies and biomarker development.
✅ In October 2025, Addex Therapeutics launched a pivotal Phase IIb trial for its small molecule therapy addressing neuropathic pain in CMT patients.
Europe: Recent Industry Developments
✅ In October 2025, Pharnext S.A. announced positive topline results from its PREMIER trial of PXT3003 for CMT1A treatment in European patients.
✅ In October 2025, the European Medicines Agency granted Priority Medicines (PRIME) designation to Affectis Pharmaceuticals' CMT treatment candidate.
✅ In October 2025, Lupin Limited entered a strategic partnership with a European biotech to develop and commercialise novel CMT therapies across EU markets.
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Market Trends & Drivers
The Charcot-Marie-Tooth Disease Market is experiencing significant growth driven by increasing disease awareness, advancements in genetic research, and growing investment in orphan drug development. Key trends include the emergence of gene therapies, development of targeted symptom management, and expansion of patient registries for clinical trial recruitment. The improving diagnostic capabilities, orphan drug incentives, and collaborative research initiatives are significantly accelerating market development.
Competitive Landscape
The market features specialized competition among biotechnology companies, pharmaceutical firms, and research organizations focusing on rare neurological diseases.
✦ Neurogene Inc. leads in gene therapy development for CMT and related inherited peripheral neuropathies.
✦ Pharnext S.A. pioneers combination therapy approaches with its lead candidate PXT3003 for CMT1A.
✦ Affectis Pharmaceuticals AG focuses on novel molecular targets for CMT treatment and symptom management.
✦ Addex Therapeutics Ltd. develops allosteric modulators for neuropathic pain in CMT patients.
✦ Lupin Limited expands its neurology portfolio with CMT therapeutic candidates and diagnostic tools.
✦ DTx Pharma Inc. advances RNA-based therapeutics for various CMT subtypes.
✦ Viking Therapeutics Inc. explores metabolic modulators for CMT-related muscle weakness and fatigue.
✦ StrideBio Inc. develops novel capsids for gene therapy delivery in CMT and other neurological disorders.
✦ Taysha Gene Therapies Inc. focuses on AAV-based gene therapies for monogenic neurological diseases, including CMT.
✦ Applied Genetic Technologies Corporation (AGTC) develops gene therapies for inherited diseases, including certain CMT subtypes.
Segmentation
By Disease Type (CMT1, CMTX, CMT4, CMT2, Others)
By Treatment Type (Physical Therapy, Occupational Therapy, Orthopedic Devices, Others)
By End-User (Hospitals, Specialty Clinics, Home Care Settings, Others)
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Regional Analysis:
⇥ North America (U.S., Canada, Mexico)
⇥ Europe (Germany, U.K., France, Italy, Spain, Rest of Europe)
⇥ Asia-Pacific (Japan, China, India, Australia, South Korea, Rest of Asia Pacific)
⇥ South America (Brazil, Argentina, Rest of South America)
⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)
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