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

WHO WE ARE

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.

CMTRF Invests in Project to Test if a Commercially Available Drug is Effective Treatment for X-linked Charcot-Marie-Tooth Disease

CMTRF has invested in a project led by Dr. Charles Abrams, a Professor in the Department of Neurology and Rehabilitation at the University of Illinois at Chicago. The study will determine if a commercially available drug can improve symptoms in mouse models of Charcot-Marie-Tooth disease type 1X-which is the second most common form of CMT.