CMTA Headlines:
We’re Community Powered!
This September, we ask you to join the CMTA in celebrating International CMT Awareness Month 2018! CMT Awareness Month is our month, our chance to promote CMT awareness, understanding and education. Learn more…
Three Major Patient Advocacy Groups Focused on CMT Research Collaborating on FDA “Voice of the Patient” Report
This CMT Awareness Month, CMTA is proud to join the Muscular Dystrophy Association as a sponsor of the Hereditary Neuropathy Foundation who will be hosting the Externally-led Patient-Focused Drug Development Meeting. Learn more…
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?
The CMTA’s STAR (Strategy to Accelerate Research)
STAR – Translational Advances
