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.

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...

CMT Research Foundation Awards $152,524 to ORYZON to test novel HDAC6 inhibitors in CMT1A

Jul 26, 2022 | CMTRF Funded Research, Research News

New project will examine impact of two selective and potent drug candidates recently discovered by ORYZON The CMT Research Foundation is delighted to announce a new project with ORYZON, a clinical stage pharmaceutical company based in Spain. Histone deacetylase (HDAC)...

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...

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...

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...

Legacy – an Anthem for Patients with CMT

May 13, 2022 | Life with CMT, News

Recording-artist/songwriter/producer Dina Fanai AKA AVALONA, released Legacy today across all platforms Legacy was written as an anthem for patients with Charcot-Marie-Tooth disease (CMT), one of the world’s most common inherited neurological diseases. CMT causes...

2022 Global CMT Research Convention—Introducing the Scientific Program Committee

May 11, 2022 | CMTRF Funded Research

As we get ready for the 2022 Global CMT Research Convention, one of the most important events is the empanelment of a Scientific Program Committee, or SPC. What does the SPC do? In general, an SPC assists in the development of the scientific content for a convention....

CMT Mothers

May 4, 2022 | Life with CMT

The CMT Research Foundation knows that moms in the world of CMT have unique challenges and trials. And we salute their tenacity, patience, and unyielding dedication. Here are the stories of two CMT moms; one whose daughter lives with CMT and another who is a patient....