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.
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.
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...
Schwann Cell-Targeted Gene Therapy Approaches to Treat CMT1A and Other Demyelinating Neuropathies
With CMTA support of over $160,000, an international team of researchers is developing genetic therapy...
