CMT2D Research
…Scientific Advisory Board, is using mouse models of several forms of CMT to determine which types may be candidates for treatment with HDAC6 inhibitors and whether HDAC6 inhibitors may be…
CMT2S Research
…using mouse models of several forms of CMT to determine which types may be candidates for treatment with HDAC6 inhibitors and whether HDAC6 inhibitors may be of therapeutic benefit across…
The CMTA Is Accelerating Research Through Gene Therapy
…is to transform it into effective treatments for people living with CMT. Given the increased feasibility and applicability of gene therapy to CMT, the CMTA hosted a Gene Therapy Workshop…
Finding Your CMT Gene
Finding Your CMT Gene – The Distant Cousin Project The effort to identify genes that cause rare diseases is more important than ever since effective treatments for genetically caused rare…
CMT1X Research
…help advance development of treatments for other types of CMT affecting Schwann cells, as a similar AAV approach can be applied to CMT1A and other subtypes of CMT1. PROJECT GOAL:…
CMTA-STAR Awards $1.1 Million for CMT1X, CMT2A Clinical Trials
…changes over shorter periods of time than self-reported measures like clinical outcome assessments so that researchers can quickly and precisely measure whether a treatment or drug had a positive impact…
CMTA-STAR Research for Axonal Forms of CMT
…to the market in the near future. The CMTA’s funding and operations focus is on translational research that will lead as directly as possible to therapeutic treatments for CMT. Axonal…
Who Inspires You to Give?
…that seeing the CMTA’s research progress and knowing that she is leaving a legacy and contributing to a treatment/cure inspires her to give. Under the CMTA’s Strategy to Accelerate Research,…
The Fall 2020 CMTA Report
…CMT Join Tribe of the Funky Feet Online CMT1A, CMT1B, CMT4B Projects Approved CMT1A Research Agenda Fuller than Ever 1A Timeline: CMTA Roadmap to Success Consensus Statement on Surgical Treatment…
The CMT&ME Study: What It’s Like to Live with CMT
…how treatment can improve patient quality of life and slow CMT progression. The study collects data directly from patients using a digital app, CMT&Me, which collects real-world data using “bring…