CMTA Strategy to Accelerate Research (CMTA-STAR) Alliance Partner Vanda Pharmaceuticals has announced that the U.S. Food...
CMTA Expands Preclinical Toolbox with New CMT1A Assay
CMTA expands its Preclinical Toolbox with a new CMT1A assay, providing researchers with a human-relevant platform to evaluate potential therapies earlier in drug development.
What 465 People with CMT1A Want Researchers to Know
Results from a patient survey conducted by CMTA, CMT France, and Nervosave Therapeutics highlight what matters most to people living with CMT1A.
Cycle Pharmaceuticals Stops Clinical Studies of Govorestat
On April 17, Cycle Pharmaceuticals announced it is halting all govorestat clinical studies, including the INSPIRE Phase III Open Label Extension and Early Access Programs.
CMTA Invests $141,000 to Study Hearing and Balance to Develop Better Clinical Trial Outcome Measures in Charcot-Marie-Tooth Disease
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.
Blood–Brain Barrier-Crossing Conjugates for Treating CMT
CMTA-funded research at Mount Sinai is studying how RNA medicines can be delivered into nerve cells in CMT, overcoming biological barriers that block treatment.
Preliminary Characterization of STEP Platform for Delivery of Genome Editing Therapy for CMT
CMTA-funded Yale researchers are testing STEP, a non-viral delivery system for genome-editing medicines in CMT.
CMTA-STAR Alliance Partner NMD Pharma Shares SYNAPSE-CMT Trial Update
New Phase 2a CMT trial results are in. NMD Pharma shares details.
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.
