Atlanta, GA (September 30, 2024) The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)* announced over the weekend at its annual Global CMT Research Convention that it has surpassed its goal of raising $10 million to fund research to find treatments and cures for CMT Type 1A.

Peter J. de Silva, CMTRF Board Chair, CMT patient and Chair of the ENDGAME Capital Campaign launched the campaign three years ago saying, “For the first time in more than 45 years, I am encouraged that the science is at a point where treatments and cures are soon to be a reality. We are closer than ever to finding treatments and cures. I can’t wait for that day.  Because we believe that the end of CMT1A is in sight, CMTRF calls the campaign ENDGAME. Moreover, CMTRF is confident that successes achieved by the research underwritten by ENDGAME will eventually benefit all types of CMT.”

“The Foundation has put the money raised to immediate work for drug development for CMT1A.  To date funds have fueled 9 projects from six companies and three top academic research centers aimed at developing or advancing therapies and cures for CMT1A,” says Cleary Simpson CEO of CMTRF. “Three of the companies have drugs that they are preparing for clinical trials: Oryzon Genomics, Augustine Therapeutics and Armatus Bio.  Another three companies hadn’t worked in CMT before; ENDGAME activated their technologies to be applied to CMT1A.”  

“I, along with many other generations of other family members, have lived with this disease since our respective childhoods,” says Susan Ruediger, Co-Chair of ENDGAME and CMTRF’s Chief Mission Officer. “It just has to stop, and we are now in a position to make that happen.” 

CMT Research Foundation (CMTRF) is a patient-led, non-profit focused on delivering treatments and cures for CMT. The foundation identifies significant obstacles or deficiencies impeding progress toward a cure and seeks out collaborators to address these issues. To date, CMTRF has funded 24 projects, of which 8 are completed. Of those 8 completed projects, 5 have clinical candidates. CMTRF’s mission to invest in promising science with high potential of leading to treatments and cures was proven effective and ground-breaking when DTx Pharma with a CMTRF- backed program as its lead candidate was acquired by Novartis for $1 billion. Founded by two patients who are driven to expedite drug delivery to people who live with CMT, the 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts. 

*Charcot-Marie-Tooth encompasses a group of inherited, chronic peripheral neuropathies that result in nerve degradation. CMT patients suffer from progressive muscle atrophy of legs and arms, causing walking, running and balance problems as well as abnormal functioning of hands and feet. CMT affects one in 2,500 people (about the same prevalence as cystic fibrosis), including 150,000 Americans and nearly 3 million people worldwide. At the moment, there is no treatment or cure for CMT.

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