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 Research Foundation Partners to Advance Study of CMT1J by Dr. Stephan Zuchner

CMTRF and the 1J Foundation have joined forces to support the development of a mouse model for CMT1J. Led by Dr. Stephan Zuchner at the University of Miami, the project aims to understand how mutations in the ITPR3 gene, which regulates calcium flow in nerve cells, contribute to CMT1J.

The CMT Research Foundation Makes Leadership Changes to Drive Next Phase of Growth; Names Peter de Silva as Board Chair

At its recent annual meeting, The CMT Research Foundation made changes in its leadership to support the next phase of the Foundation’s growth.

DTx Pharma Acquired by Novartis for CMT1A Therapy

This is one of CMTRF’s earliest investments


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