Tracking CMT4J from the Inside Out: What MRI Scans Are Showing So Far With CMTA...
Accelerating Genetic Therapy for CMT4B1: A CMTA Research Update
With CMTA’s support, researchers at San Raffaele Scientific Institute in Milan, Italy, led by Alessandra...
AAV9-Mediated Gene Therapy to Treat Inherited CMT Neuropathies With Aberrant Myelin
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...
CMTA’s $240K Investment Targets Gene Replacement Therapy in CMT4 Subtypes
CMTA announced a $240,000 investment AAV-based gene replacement for CMT4A, with potential implications for CMT4B1 and CMT4D.
