This CMTA-funded study is testing a gene therapy approach for CMT4B1, a rare form of CMT that affects the myelin around nerves. Early results are encouraging, showing potential to restore nerve health by delivering a working copy of the affected gene.
Measurement of muscle fat fraction in calf and thigh muscle in patients with CMT4J
With CMTA support of $149,443, researchers at Elpida Therapeutics are investigating muscle fat fraction (MFF)...
Gene Replacement Therapy for CMT4C IND-Enabling Studies
CMTA and families are driving Project Foresee to bring genetic therapy for CMT4C into clinical trials. Learn how this effort is moving toward patients.
Characterization of Rat Models Expressing Human Mutations of CMT4A
Gene Replacement Therapy for CMT4A Neuropathy in Rodents
With CMTA support of $248,950, researchers at the University of Texas Southwestern, led by CMTA’s...
