Medication Study

…lithium, and pyridoxine. Overall, Weimer and Podwall conclude that treatment with vincristine poses an “unacceptable risk to patients with known or possible CMT1A.” They further conclude that the use of…

Type 1 CMT Gene Therapy Project Now Underway

…CMT (type 1 and most type 4).” “Gene therapy continues to show promise for the treatment of neuromuscular disease,” said Amanda Haidet-Phillips, Ph.D., one of the MDA’s scientific portfolio directors….

Type 1 CMT Gene Therapy Project Now Underway

…CMT (type 1 and most type 4).” “Gene therapy continues to show promise for the treatment of neuromuscular disease,” said Amanda Haidet-Phillips, Ph.D., one of the MDA’s scientific portfolio directors….

CMTA Board of Directors

…as leading the charge for development of treatments. Bernard Coulie Bernard is the CEO and President of Pliant Therapeutics, a NASDAQ-listed biopharmaceutical company focused on developing novel treatments for rare…

CMT1A Research Breakthrough!

A paper published December 4th on the Journal of Clinical Investigation (JCI) website reveals an exciting potential treatment for patients with Charcot-Marie-Tooth (CMT), the progressive neuromuscular disease that affects more…