CMT Research Foundation
Patient-led non-profit organization focused on accelerating development of treatments and cures for Charcot‑Marie‑Tooth (CMT) disease by funding translational research, convening stakeholders, and connecting academic and industry partners.
Industries
N/A
Services
Research grant funding and project management
Provides targeted funding, milestone management, and scientific oversight for preclinical and translational projects focused on CMT.
Global CMT Research Convention
Annual multi‑day meeting that convenes scientists, clinicians, industry, investors and patients to share data and stimulate collaborations.
Ambassador and volunteer programs
Volunteer network that supports fundraising, awareness, and local outreach for research initiatives.
Patient resources and trial information
Provides newly‑diagnosed information packages, clinical trial listings and guidance for patients seeking research participation.
Research grant funding and project management
Provides targeted funding, milestone management, and scientific oversight for preclinical and translational projects focused on CMT.
Global CMT Research Convention
Annual multi‑day meeting that convenes scientists, clinicians, industry, investors and patients to share data and stimulate collaborations.
Ambassador and volunteer programs
Volunteer network that supports fundraising, awareness, and local outreach for research initiatives.
Patient resources and trial information
Provides newly‑diagnosed information packages, clinical trial listings and guidance for patients seeking research participation.
Expertise Areas
- Translational research funding and portfolio management
- Gene therapy development for peripheral neuropathies
- Oligonucleotide and RNA therapeutic delivery to peripheral nerves
- Patient‑derived cellular models and high‑throughput screening
Key Technologies
- Lipid nanoparticle delivery
- Viral vector gene therapy
- Antisense oligonucleotides (ASOs)
- Oligonucleotide conjugation and receptor‑mediated delivery