What happens when scientists flip a hidden switch inside nerve cells? A CMTA-funded $281,339 study is testing whether this discovery could finally bring new treatment options for people with CMT1A.
CMTA Invests $281,339 to Advance Treatments for CMT1A
The newly funded study will test whether molecules originally in development as cancer therapeutics can also be used to lower PMP22 and improve nerve health in a model of CMT1A.
Modulating TEAD1 Activity in Charcot-Marie-Tooth Disease Type 1A
With CMTA support of $281,339, researchers at Albany Medical College, led by Sophie Belin, PhD, and Yannick Poitelon, PhD, are exploring the newly discovered TEAD1 pathway in Schwann cells that may regulate PMP22, the gene at the root of CMT1A.
FDA Meeting Could Pave the Way for First CMT Treatment
Applied Therapeutics secures FDA meeting this fall on its plan to submit govorestat for CMT-SORD. And what’s this about a free SORD test?
This Genetic Tape Could Silence the Cause of CMT2E
What happened when CMTA-supported scientists at the University of Miami applied a piece of genetic tape to the gene mutation that causes CMT2E?
Gene Editing for CMT Is Getting Real: The Future Just Shifted Gears
CMTA-supported scientists are using CRISPR to go after the genetic root of CMT1A and CMT1B. Early results are changing what’s possible. See how this research is shifting from bold ideas to future realities.
What If We Could Measure CMT4J Getting Worse Before It Feels Worse?
Tracking CMT4J from the Inside Out: What MRI Scans Are Showing So Far With CMTA...
CMTA-STAR Alliance Partner Doses First CMT2S Patient
CMTA-STAR Alliance Partner Doses First CMT2S Patient
CMTA and INC Forge Groundbreaking Alliance to Accelerate Comprehensive Research Across All Charcot-Marie-Tooth Disease Types
What happens when the world’s largest CMT funder joins forces with the leading clinical research network? A new era begins. Inside: the alliance changing everything.
Augustine Therapeutics – AGT-100216
The Phase I trial will focus on safety, tolerability, and pharmacokinetics (how the drug moves...
