New, Potentially Treatable, Type of CMT Discovered

A team led by Dr. Stephan Züchner at the University of Miami has discovered a new type of Charcot-Marie-Tooth (CMT) disease that may be treatable with drugs already approved for other diseases.

CMT2E Gene Therapy Project Approved

The CMTA Board of Directors approved a $265,000 research project for CMT2E that aims to advance CMT2E gene therapy by demonstrating positive results for axonal forms of CMT affecting motor neurons.

CMTA Board Approves CMT1B Project

The CMTA Board of Directors approved a new research project aimed at developing biomarkers for CMT1B to aid in the collection of natural history data critical to clinical trials.

CMTA Funds Cutting-Edge Gene Editing Studies for Type 2 CMT

Drs. Bruce Conklin and Luke Judge will use a $653,000 grant from the CMTA to develop the gene-editing technique known as CRISPR for application to CMT2A, 2E and 2F.

Another $500,000 Pledge to Type 2 Research!

Bruce Chizen, a special advisor to the Board of Directors, will match donations to CMT2 research on a one-to-one basis, up to $500,000.

New Gene Therapy, Type 1/Type 4 Research Projects Approved

The CMTA today announced another $335,000 in grants to top CMT scientists leading the field in CMT Type 1 research. The grants cover work on types 1A, 1B and 1X and other demyelinating forms of CMT, including type 4.

The CMT&ME Study: What It’s Like to Live with CMT

There’s been little research to date into how CMT affects

New Gene Therapy Development Program for CMT2A

Passage Bio, a genetic medicines company developing AAV-delivered gene therapies, today announced a gene therapy development program for Charcot-Marie-Tooth Neuropathy Type 2A (CMT2A).

$1 Million Gift Pledged to CMT Type 2 Research!

Bob and Gail Buuck are big believers in “impact giving,” They have pledged a $1 million gift to advance CMT Type 2 research.

Type 1 CMT Gene Therapy Project Now Underway

CMTA and MDA Co-Fund Proof-of-Concept Study Using AAV Vector to