News & Stories

See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.

Italian Research Group Completes CMT Research Foundation-Funded Project, Results Encourage Further Work in New Therapy for Adult and Elderly Patients with CMT1A/CMT1B

Jan 10, 2023 | CMT Research Updates, CMTRF Funded Research, Research News

The CMT Research Foundation is pleased to announce that Dr. Maurizio D'Antonio's research team at San Raffaele Scientific Institute in Milan, Italy has completed their CMT Research Foundation-funded project investigating the role of a protein called Gadd34 in a mouse...

2022 at CMTRF

Jan 9, 2023 | CMT Research Updates, CMTRF Funded Research, Press Releases

Dear Friends of CMTRF, In preparing for another busy year at CMTRF, I took some time recently to look back at the accomplishments of 2022. I am immensely proud of what our team has achieved (summarized below) and I believe it underscores how CMTRF adds significant...

Muscular Dystrophy Association and CMT Research Foundation Announce Joint Research Study Grant

Dec 14, 2022 | CMTRF Funded Research, Press Releases

(New York) December 14, 2022 The Muscular Dystrophy Association (MDA) and CMT Research Foundation (CMTRF) announce joint funding for a study by Alessandra Bolino, PhD, at San Raffaele Hospital (Ospedale San Raffaele), Italy, on restoring membrane trafficking in...

CMT Research Foundation partners with Samsara Therapeutics to develop a novel class of drugs in CMT1A

Oct 13, 2022 | About CMT, CMT Research Updates, CMTRF Funded Research, Research News

The CMT Research Foundation is pleased to announce a new project with Samsara Therapeutics, a US/UK-based biotech company that is developing a novel class of drugs for CMT1A. CMT1A is caused by duplication of a stretch of DNA that includes the PMP22 gene, and people...

The CMT Research Foundation Invests Over $500,000 to Find a Single Gene Therapy for Possibly Every Form of CMT1B

Jul 18, 2022 | CMT Research Updates, CMTRF Funded Research, Research News

CMT1B is one of the most common forms of CMT. In CMT1B, the part of the nervous system that is dysfunctional is the myelin sheath of the peripheral nerves. Created by cells called Schwann cells, myelin wraps around the parts of the nerves that facilitate rapid...

CMT Research Foundation Awards Grant to Nationwide Children’s Hospital’s Dr. Afrooz Rashnonejad to Develop Gene Therapy to Cure or Alleviate CMT1B

Jul 18, 2022 | Press Releases

ATLANTA (July 18, 2022) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease*, has awarded a grant of over $500,000 to find a single gene therapy for every form of CMT1B. The award was made...

Christopher Austin to Keynote CMT Research Foundation’s 2022 Global CMT Research Convention

Jul 12, 2022 | Press Releases

Christopher Austin, Founding Director of the National Center for Advancing Translational Sciences, to Keynote CMT Research Foundation’s 2022 Global CMT Research Convention ATLANTA (July 12, 2022) Christopher Austin, M.D., CEO of Vesalius Therapeutics, CEO-Partner of...

Project Testing PIKfyve Inhibitor in CMT4B1 Clears First Hurdles

Jul 11, 2022 | About CMT, CMT Research Updates, CMTRF Funded Research, Research News

CMTRF is happy to report that a CMTRF-funded project designed to test a PIKfyve inhibitor in CMT4B1, a particularly devastating form of the disease, has passed the first stage and is moving on to the next stage, a preclinical trial in CMT4B1 model mice. Before the...

CMT Research Foundation emerging as a leader at BIO International Convention

Jun 29, 2022 | CMT Research Updates

The CMT Research Foundation meets with more than 30 companies at BIO and is recognized as a leader in patient driven research.

Shift Pharmaceuticals Completes CMT Research Foundation-Funded Project, Will Continue Development of Gene Therapy for CMT1A

May 4, 2022 | CMT Research Updates, CMTRF Funded Research, Research News

The CMT Research Foundation is pleased to announce that Shift Pharmaceuticals (Shift) has successfully completed their CMT Research Foundation-funded RNA-based therapy project. Shift created and tested a library of novel molecules designed to reduce the expression of...