Charcot-Marie-Tooth Association

CMTA Headlines:

The Summer 2018 CMTA Report

In this issue: New Alliance Partners, Pulmonary Care, Hearing Loss, Smart Homes and more …

Regenacy and CMTA Announce Strategic Partnership to Evaluate Ricolinostat, a Selective HDAC6 Inhibitor

The CMTA and Regenacy Pharmaceuticals have announced a collaboration to validate the role of HDAC6 in multiple forms of Charcot-Marie-Tooth (CMT) disease and evaluate the efficacy of ricolinostat, a selective HDAC6 inhibitor, in animal models. The CMTA’s STAR Advisory Board member David Herrmann, M.B.B.Ch., has also joined Regenacy’s Scientific Advisory Board. Learn more…

CMTA Announces Strategic Partnership with Acceleron Pharma to Advance CMT Treatment Options

The CMTA announced a strategic partnership with Acceleron Pharma, a Cambridge-based biopharmaceutical company dedicated to developing medicines to treat serious and rare diseases, including CMT. The CMTA’s Strategy to Accelerate Research (STAR) connects top CMT clinicians and academic researchers with pharmaceutical partners committed to developing treatments, and ultimately, a cure for CMT. Strategic alliances with corporate partners like Acceleron support innovative drug development with a goal to rapidly deliver therapies to CMT patients. Learn more…

The CMTA Leads the Way in Research

For the past decade, the CMTA has led the way in research worldwide with its Strategy To Accelerate Research (STAR). Our mission is to support the development of new drugs to treat CMT, to improve the quality of life for people with CMT, and, ultimately, to find a cure. We thank our community members who have been with us every step of the way! To learn more about STAR, please visit www.cmtausa.org/research/our-star-strategy.

CMT1A Research Breakthrough!

Scientists at Ionis Pharmaceuticals, in collaboration with the Charcot-Marie-Tooth Association, have identified a promising early-stage therapeutic approach with antisense technology for CMT Type 1A (CMT1A), the most common form of the disease, accounting for half of all cases. Read more …

Breakthroughs to a Cure

Studies in two laboratory models of CMT1A not only stopped progression of the disease, but also showed improvement of some symptoms. To deliver the first treatments for CMT1A and other types of CMT, we need funding for clinical trials and further drug development.

What is CMT?

Charcot-Marie-Tooth, or CMT, is the most commonly inherited peripheral neuropathy and affects an estimated 2.8 million people.

Find Your Local Branch

The CMTA has over 80 Branches across North America to support people with CMT, raise awareness, and fund research. Connect with your nearest Branch today!

Fundraise for Our Future

Without fundraising and community volunteers, the CMTA would not be the leading force in CMT Research that it is today. Are you ready to make an impact?

Shop Our Store

Visit our store to see some of the most fun and fashionable ways to raise awareness!

The CMTA’s STAR (Strategy to Accelerate Research)

The CMTA’s vision is a world where patients with CMT neuropathies have therapies available to them to manage or modify their disease. That vision is poised to become reality as STAR pushes the research forward at an ever-increasing speed.

STAR – Disease Project Team Updates

The CMTA’s research is done in teams consisting of academic labs and clinical centers sponsored by the CMTA (STAR members), working together and with alliance partners in consortium team efforts. STAR Teams are sponsored by the CMTA via a rapid process of expert review, following invitation of proposals for targeted translational efforts. All sponsorship aims to directly aid the advancement of CMT therapies.