Can we fix the genes that cause CMT? CMTA’s Dr. Katherine Forsey breaks down the science behind genetic therapies and what’s heading toward clinical trials.
CMTA-STAR Alliance Partner Doses First CMT2S Patient
CMTA-STAR Alliance Partner Doses First CMT2S Patient
Augustine Therapeutics Begins New CMT Trial
CMT news out of Belgium: Augustine Therapeutics has officially dosed the first participant in a...
Augustine Therapeutics – AGT-100216
The Phase I trial will focus on safety, tolerability, and pharmacokinetics (how the drug moves...
CMTA Presents New Genetic Landscape Analysis at The 2025 Annual Meeting of the Peripheral Nerve Society
The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic funder of Charcot-Marie-Tooth (CMT) disease research, presented a...
CMTA-INC Alliance Announces 2025 Inherited Neuropathies Fellowship Recipient
The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic funder of Charcot-Marie-Tooth disease (CMT) research, today announced...
CMTA-INC Alliance Announces 2025 Inherited Neuropathies Fellowship Recipient: Orthopedic Surgeon to Focus on Improving Outcomes for People with CMT
The CMTA–INC Alliance announced the 2025 Inherited Neuropathies Fellowship recipient, supporting clinical research focused on corrective foot and ankle care and surgical outcomes for people with CMT.
Long Read Sequencing in Undiagnosed Axonal CMT Patients
The answer is hiding in your DNA. For some, it hasn’t been found yet. This CMTA-funded study uses long-read sequencing to uncover genetic causes of CMT that standard tests may miss.
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.
