The CMT Research Foundation: Delivering Treatments and Cures for Charcot-Marie-Tooth


The CMT Research Foundation is a patient-led, non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease. CMT affects one in 2,500 people (about the same prevalence as multiple sclerosis), including 150,000 Americans and more than 3 million people around the world.

We have one single mission: to raise funds to invest in science that will lead to treatments and cures for CMT. Our impact goes beyond simply making investments. We identify key problems or gaps in progress to a cure, then aggressively find partners to attack the problems. We introduce CMT to the greater drug development community, something that has rapidly increased the number of experts working to solve the disease.

We fund projects with the highest likelihood of leading to clinical trials and approved treatments in the near term. All investments are gated, meaning projects need to achieve milestones defined for each phase of work before receiving funding for the next. Every contract includes a return on the CMT Research Foundation’s initial investment which is then reinvested in new projects.

CMT Convention Logo

Registration is Now Open for the CMT Reseach Foundation’s 2024 Global CMT Research Convention

The 2024 CMT Research Convention will examine and discuss advances and remaining challenges in CMT drug development to expand collaboration and accelerate research dedicated to developing treatments for all types of CMT.


CMTRF at the 2024 American Society of Gene and Cell Therapy Conference

We are thrilled to see that two CMTRF-funded projects were selected to present their advancements on their CMT gene therapy projects at the ASGCT conference, the premier conference for emerging genetic therapies.

Gene Therapy

Gene therapy is a technique to modify or control gene expression to treat or cure diseases. For CMT, gene therapy can correct genetic mutations by replacing, adding, silencing, or modifying affected genes. This approach has succeeded in treating other genetic diseases like Spinal Muscular Atrophy. Targeting these mutations with gene therapy is a potential treatment for many CMT subtypes.


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Media Inquiries

George Simpson


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