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.

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...

Dr. Arthur Suckow, DTx Pharma Inc. – FALCON

Nov 28, 2022 | CMTRF Funded Research, Drug Development

One of the first research teams the CMT Research Foundation (CMTRF) funded was DTx Pharma, Inc., and the project focused on delivery of genetic medicine to the myelinating Schwann cells of the peripheral nerves - a major scientific challenge to effectively treating...

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...

CMT Research Foundation and Samsara Therapeutics Partner on a Novel Therapeutic Approach for CMT1A with the Potential to Reach Clinical Trials

Oct 12, 2022 | CMTRF Funded Research, Press Releases, Research News

ATLANTA (October 13, 2022) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease*, has invested in Samsara Therapeutics Inc., a biotech company focusing on discovering and developing...

ORYZON collaborates with the CMT Research Foundation in the US

Jul 26, 2022 | CMTRF Funded Research, Press Releases, Research News

CMTRF to provide funding for in vivo efficacy tests of Oryzon’s HDAC6 inhibitors in Charcot-Marie-Tooth preclinical model MADRID, SPAIN and Atlanta, July 26, 2022 Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company...

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...

A New Trial for CMT – When Science Moves Quickly

May 18, 2022 | Drug Development, News

By Keith Fargo, Ph.D., Chief Scientific Officer, CMT Research Foundation The pace of science can seem excruciatingly slow. It can take many years, or even decades, for new ideas to result in clinical trials. Every so often, though, the stars align and a new...

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, News, Press Releases, 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...