CMTA

What CMTA does

CMTA (Charcot-Marie-Tooth Association) is a 501(c)(3) nonprofit organization founded in 1983 and headquartered in Glenolden, Pennsylvania, dedicated to advancing treatments and patient support for Charcot-Marie-Tooth disease (CMT), a hereditary peripheral neuropathy affecting more than 3 million people worldwide. The organization operates four interlocking platforms: (1) CMTA-STAR (Strategy To Accelerate Research), a research funding and biotech alliance program that has invested over $33 million in CMT research since 2008 and maintains a public pipeline of therapies in development; (2) a global network of CMTA Centers of Excellence providing multi-disciplinary CMT clinical care, with the first international site opened in Dublin, Ireland in October 2025; (3) the Patients as Partners in Research patient registry, which feeds the CMTA-INC Alliance dataset covering over 7,000 enrolled patients across 20 clinical sites to support natural history studies and clinical trial readiness; and (4) a community engagement layer including local Branches, Camp Footprint (serving 160+ youth annually in Oregon and Pennsylvania), the new Footprint Fellowship leadership program, and youth councils.

CMTA's core technical assets are the CMTA-INC Alliance patient dataset and the CMTA-STAR Preclinical Toolbox (research models and tools). The organization partners with biotech and pharmaceutical companies developing CMT therapies, including Applied Therapeutics (govorestat for CMT-SORD, with an FDA NDA meeting granted), NMD Pharma (NMD670 in the Phase 2 SYNAPSE-CMT trial with 81 participants, data expected early 2026), Alesta Therapeutics (CMT2 gene therapy), Nervosave Therapeutics (CMT1A gene therapy), EverTree Bio, and Kylix Bio, alongside academic research collaborations at Yale, Mount Sinai, University of Wisconsin-Madison, UT Southwestern, Cyprus Institute of Neurology and Genetics, and Michigan Medicine.

Its business model relies entirely on donations and charitable contributions as a 501(c)(3), supplemented by co-funded grants with partners including MDA, NIH, and CIRM. CMTA has demonstrated catalytic funding leverage, with $1.15 million in CMTA-supported research leading to nearly $20 million in subsequent government grants. The organization reaches its audience through digital channels (website, podcast, social media), in-person events (Walk & Cycle 4 CMT fundraising events, the annual CMTA Patient & Research Summit), and direct community programs, and is governed by a Board of Directors chaired by Wendy Arnone, with CEO Sue Bruhn, PhD leading operations.

Name

CMTA

Legal name

Charcot-Marie-Tooth Association

Website

https://cmtausa.org

Company type

Private

Founded year

1983

Operating status

Operating

Headcount range

11–50 employees

Short description

CMTA (Charcot-Marie-Tooth Association) is a 501(c)(3) nonprofit organization that funds Charcot-Marie-Tooth disease research and provides patient support services. It serves CMT patients and families globally through the CMTA-STAR research program, a network of Centers of Excellence, and community programs.

Ownership category

akta.pro rank

Product category

Rare Disease Patient Advocacy

NAICS

Other Individual and Family Services (624190), Individual and Family Services (6241)

SIC

Services-Health Services (8000)

akta.pro primary industry

Post-Acute Care Networks & Care Management Organizations (HLAFAFAN)

akta.pro secondary industries

Community Mental Health Centers (CMHCs) & Public Behavioral Health Providers (HLAFACAI), CMC & Pharmaceutical Development (Formulation/Process/Analytical Development) (HLAIALAD)

Keywords

• Rare disease research
• Patient advocacy services
• Neuromuscular disorder support
• Nonprofit health organization
• Clinical research funding

Headquarters

HQ city

Glenolden

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2C

Offering type

Services

Cost components

Personnel, Operations, Technology or R&D, Marketing or Sales, Others

Revenue model

1. Donations and Charitable Contributions: CMTA operates as a 501(c)(3) nonprofit organization funded primarily through donations from individuals, families, and supporters. The organization has invested over $33 million in CMT research since 2008.

Go-to-market motion1 record

Distribution channels5 records

Marketing channels9 records

Core offering

CMTA (Charcot-Marie-Tooth Association) is a 501(c)(3) nonprofit that funds and accelerates research toward treatments for Charcot-Marie-Tooth disease (CMT) through its CMTA-STAR program. It operates a global network of CMTA Centers of Excellence providing expert patient care, runs community support programs including Camp Footprint and local Branches, and maintains the Patients as Partners in Research registry to connect patients with clinical trials.

Product overview

CMTA (Charcot-Marie-Tooth Association) is a nonprofit organization focused on accelerating CMT research and empowering patients. The organization operates CMTA-STAR (Strategy To Accelerate Research) as its core research program, funding projects and maintaining a pipeline of CMT treatments in development. Patient engagement is facilitated through the Patients as Partners in Research registry and the CMTA-INC Alliance with the Inherited Neuropathy Consortium. CMTA provides care and support through a global network of Centers of Excellence, local Branches, and community programs including Camp Footprint and the Footprint Fellowship for youth. Educational resources include the Simply CMT Blog, Simply CMT Podcast, Publications, a Gadget Store, and the CMTA-STAR Preclinical Toolbox for researchers. The organization also hosts events like Walk & Cycle 4 CMT and an annual Patient & Research Summit.

Differentiator

Problem solved

Functional benefit

Products and services

• CMTA-STAR (Strategy To Accelerate Research)

Named customers2 records

Segments4 records

Ideal customer profiles4 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

14 partnerships are on record, tiered core and minor.

• Inherited Neuropathy Consortium (INC)coreStrategic or Co-development PartnerThe CMTA-INC Alliance is a strategic partnership to accelerate clinical research and trial readiness in CMT. The INC maintains the largest CMT patient data set with over 7,000 patients across 20 clinical sites, supporting natural history studies for all forms of CMT.
• Applied TherapeuticscoreStrategic or Co-development PartnerCMTA-STAR Alliance Partner developing govorestat (AT-007) for CMT-SORD. The FDA granted a meeting to discuss NDA submission. CMT-SORD is caused by mutations in the SORD gene leading to sorbitol buildup toxic to peripheral nerves.
• Nervosave TherapeuticscoreStrategic or Co-development PartnerCMTA-STAR Alliance Partner, a France-based biotech company developing a genetic therapy for CMT1A offering long-lasting benefits through one-time localized injections. CMTA supported patient survey with over 200 responses.
• Alesta TherapeuticscoreStrategic or Co-development PartnerCMTA-STAR Alliance Partner focused on developing therapies for rare diseases including CMT2 subtypes. Their therapy candidate ALE2 targets mutations in tRNA synthetase genes linked to CMT2 including GARS1 in CMT2D.
• NMD PharmacoreStrategic or Co-development PartnerCMTA-STAR Alliance Partner conducting Phase 2 SYNAPSE-CMT clinical trial evaluating NMD670 in CMT types 1 and 2. Trial fully enrolled with 81 participants across US and Europe sites. Data expected in early 2026.
• Cyprus Institute of Neurology and GeneticscoreStrategic or Co-development PartnerJoint funding of $299,992 from CMTA and MDA supporting Dr. Kagiava's nanoparticle-based delivery research for CMTX1 gene therapy targeting Schwann cells.
• Muscular Dystrophy Association (MDA)coreStrategic or Co-development PartnerMDA co-funded nanoparticle-based delivery research with CMTA ($299,992 total) for developing gene-based treatments targeting Schwann cells in CMTX1.
• Shark Tooth BiominorStrategic or Co-development PartnerCombined support of $102,968 from CMTA, Shark Tooth Bio, and the National Research Council Canada for Dr. Umar Iqbal's peptide-LNP delivery research targeting Schwann cells in CMT1A.
• National Research Council Canada (NRC)minorStrategic or Co-development PartnerCombined support of $102,968 from CMTA, Shark Tooth Bio, and the National Research Council Canada for peptide-LNP delivery research for CMT1A.
• Yale UniversitycoreStrategic or Co-development PartnerCMTA supported $66,000 research at Yale (Dr. Jiangbing Zhou) developing STEP platform for delivering genome-editing medicines to peripheral nerve cells in CMT.
• Michigan Medicine - Kresge Hearing Research InstituteminorStrategic or Co-development PartnerCMTA invested $141,000 for Dr. Gabriel Corfas's research on hearing and balance biomarkers as outcome measures for CMT1A clinical trials.
• Icahn School of Medicine at Mount SinaicoreStrategic or Co-development PartnerCMTA supported $54,051 research at Mount Sinai (Dr. Yizhou Dong) developing blood-nerve barrier-crossing conjugates for delivering RNA medicines to nerve cells in CMT.
• University of Wisconsin-MadisoncoreStrategic or Co-development PartnerInternational research team led by Dr. John Svaren developing genetic therapy approaches for CMT1A targeting Schwann cells with CMTA support of over $160,000.
• University of Texas Southwestern Medical CentercoreStrategic or Co-development PartnerInternational research team led by Dr. Stephen Gray developing gene replacement therapy for CMT4A with CMTA support of over $160,000.

Scale indicators6 records

Recent moves6 records

Expansion highlights6 records

Broad incumbents

• Muscular Dystrophy Association (MDA): Large US neuromuscular disease nonprofit that co-funds CMT research with CMTA (e.g., $299,992 joint grant with Cyprus Institute). MDA is a much broader rare disease organization but overlaps on neuromuscular research funding and patient services, making it a directly comparable advocacy and research funding peer.
• National Organization for Rare Disorders (NORD): Umbrella rare disease advocacy organization of which CMTA is a Platinum Member (2026). Provides shared advocacy, research infrastructure, and policy representation across 300+ rare disease patient organizations — directly comparable mission layer at broader scale.
• Cystic Fibrosis Foundation: The gold-standard rare disease patient advocacy and research funder, having pioneered the venture philanthropy model that funds biotech drug development. Provides a long-horizon benchmark for CMTA's research-funding strategy and demonstrates how a disease-specific nonprofit can scale impact.
• ALS Association: Large US nonprofit funding ALS research and providing patient services through a national chapter network. Operates a research grant program, certified center network, and biotech partnerships — broadly comparable in structure to CMTA's Centers of Excellence, Branches, and CMTA-STAR program.

Direct peers

• CMT Research Foundation: Disease-specific nonprofit exclusively focused on funding Charcot-Marie-Tooth research toward treatments and a cure. Directly competes with CMTA for CMT-specific donor dollars, research grants, and biotech partnerships. Highly comparable mission, scope, and revenue model.
• Hereditary Neuropathy Foundation (HNF): Nonprofit dedicated to finding treatments and cures for inherited neuropathies including CMT. Operates a patient registry (GRIN), funds research, and provides patient education — closely paralleling CMTA's CMTA-INC Alliance and Patients as Partners in Research platform.
• Friedreich's Ataxia Research Alliance (FARA): Patient advocacy nonprofit advancing research for Friedreich's ataxia, another rare inherited neurological disease. Operates a patient registry (FA Global Patient Registry), funds translational research, and partners with biotech/pharma on therapy development — a near-identical operating model to CMTA in a parallel rare disease.
• Cure SMA: Disease-specific nonprofit funding spinal muscular atrophy research and supporting affected families. Has successfully advanced multiple FDA-approved therapies through coordinated research funding and patient registry programs. Closely comparable model demonstrating the trajectory CMTA aims to follow.
• Parent Project Muscular Dystrophy (PPMD): Nonprofit focused on Duchenne muscular dystrophy that funds research, runs a patient registry, and partners with biotech on gene therapy and other therapeutics. Demonstrates a similar stakeholder model to CMTA in the neuromuscular space and competes for overlapping research and donor mindshare.
• Huntington's Disease Society of America: Patient advocacy nonprofit supporting families affected by Huntington's disease, an inherited neurological condition with similar genetic and progressive characteristics to CMT. Operates Centers of Excellence, supports research, and runs a patient registry — closely paralleling CMTA's structure.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/cmtausa
• YouTubeyoutube.com/user/CMTAssociation
• Facebookfacebook.com/CMTAssociation
• LinkedInlinkedin.com/company/charcot-marie-tooth-association
• Instagraminstagram.com/cmtausa

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds

Investors

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M&A

Investments2 records

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Frequently asked questions about CMTA