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.

How to Answer: “What is CMT”

Dec 20, 2022 | Life with CMT

By Kenneth Raymond As a patient and an advocate and a student of the condition, I am often asked, “What exactly is CMT?”. My response is usually along the lines of: “CMT stands for Charcot-Marie-Tooth disease, a rare inheritable neuromuscular peripheral neuropathy...

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...

Why I Fundraise

Nov 21, 2022 | Life with CMT

My name is Anna and I have CMT1A. I fight for CMT to raise awareness to help others. I fight for CMT to inspire those living with CMT to feel less daunted about the future I fight for CMT to support research for treatments. I fight for CMT to help find a cure. I am...

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...

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.

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...

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....