CMTA Headlines:
Patients as Partners in Research: New Opportunities to Participate
The goal of Patients as Partners in Research is to advance the work of our Strategy to Accelerate Research (STAR) and enroll the patient community in the critical work of furthering the development of treatments and ultimately a cure for CMT. Read more…
Type 1 CMT Gene Therapy Project Now Underway
CMTA and MDA Co-Fund Proof-of-Concept Study Using AAV Vector to Deliver Gene Replacement Therapy in X-linked Charcot-Marie-Tooth Disease.
Research Project Identifies New Types of CMT
Dr. Stephan Züchner at the University of Miami is spearheading an ambitious project to identify new disease-causing mutations in patients.
The Winter 2018/2019 CMTA Report
New Year, New Initiatives for the CMTA! In addition to our regular features, read how the CMTA is accelerating research through gene therapy and much more.
The CMTA Is Accelerating Research Through Gene Therapy
The CMTA looks forward to a time when doctors are able to use genetic therapies to treat the root cause of CMT rather than prescribing medications or recommending surgery. We are already envisioning the possibilities that gene therapy holds for our community of 2.8 million people worldwide living with CMT. Learn more here …
STAR: The Webinar
CMTA board chairman Gilles Bouchard shares an overview of the amazing treatment-focused progress the CMTA has made over the past 10 years while highlighting STAR’s current breadth of projects, breakthroughs, and partnerships. Listen in to learn how the CMTA is using cutting-edge technologies, including stem cells, CRISPR, gene editing, gene therapy, biomarkers, high-throughput screening and axon regeneration to accelerate CMT research. View the recorded webinar at www.cmtausa.org/webinars.
The Warfield Family Makes a Difference
Do you stumble and fall? Do you have very high arches? Are your hands and ankles weak? For those of us with Charcot-Marie-Tooth (CMT), these questions represent our reality. And, for the Warfield family, this reality means three generations of family members enduring CMT. Read their inspiring story!
Progress through Partnerships – Annual Report 2017
The CMTA is pleased to announce the publication of our 2017 Annual Report “Progress Through Partnerships”. Dedicating the past decade to expediting CMT research breakthroughs, the CMTA has made great strides toward treatments and a cure by bringing together researchers, pharmaceutical and biotechnology companies, and patients. Looking back, we believe 2017 stands out as one of the most pivotal years in our journey! Please read the CMTA Annual Report 2017 here.
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…
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
