CMTA is supporting a two-year research project examining hearing and balance signaling to strengthen outcome measures in future CMT clinical trials.
CMTA Addresses the Critical Delivery Challenge Underlying CMT Treatment Development through Four Key Investments
CMTA announced $523,000 in funding for four research projects focused on solving one of the biggest challenges in CMT treatment development: delivering therapies to peripheral nerve cells. By investing in genome-editing transport, blood-nerve barrier crossing, Schwann cell targeting, and nanoparticle systems, CMTA is working to ensure treatments can reach the cells where they are needed.
When Early Testing Shows a Program Is Worth Continuing
Some research ideas reach a point where early testing answers an important question: Is this work worth continuing? For EverTree Bio, that moment came through early work using the CMTA Preclinical Toolbox.
CMTA Invests $100,000 in EverTree Bio to Support Cutting-Edge Therapeutic Research for CMT1A
CMTA announces a $100,000 investment in EverTree Bio to support continued development of an early-stage therapeutic program for CMT1A. The investment builds on early testing through the CMTA Preclinical Toolbox and reflects CMTA’s role in keeping strong CMT-focused research moving when it might otherwise stall.
ACT-CMT: The Largest-Ever Study of CMT Opens [Casting] Call for More Participants
The largest CMT1A study is expanding. New participants needed, including healthy volunteers. You can move this research forward.
ENCell Announces Phase 1b/2a Stem Cell Trial for CMT1A
Could stem cells change the path of CMT research? ENCell announces stem cell clinical trial for CMT1A.
What Happens When Scientists Flip the Switch on PMP22?
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.
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.