Cooper’s first day at Camp Footprint was also the first day he learned what it's like NOT to have to explain his CMT when meeting other kids.
CMTA Unveils Updated Logo
The CMTA’s new logo, which we are revealing for the first time in the Spring 2019 issue of The CMTA Report, is only the third version in our 35-year history.
2019 Spring CMTA Report
Clinical Trials: A Primer! In addition to our regular features, read about “A Clinical Trial for Caroline,” fast tracking and more.
A Cure for Caroline and Other CMT Stories
Caroline's journey with a rare and serious form of CMT began when she was just 9 months old. Today, a cure for Caroline may open the door to treatments and a cure for CMT!
Type 1 CMT Gene Therapy Project Now Underway
Mutations in the gene coding for the gap junction beta-1 protein (GJB1), also known as connexin 32 (Cx32), are associated with CMT1X. Dr. Kleopa will test whether delivery of the Cx32 gene using an adeno-associated virus (AAV) vector can improve symptoms in a mouse model of CMT1X.
CMTA-Funded Research Database Identifies New Mutations
While researchers have identified more than 90 mutated genes that cause CMT, approximately 50 percent of CMT2 patients do not yet have a definitive genetic diagnosis. Dr. Stephan Züchner at the University of Miami is working to change that.
Three Major Patient Advocacy Groups Focused on CMT Research Collaborating on FDA “Voice of the Patient” Report
This CMT Awareness Month, the CMTA is proud to join the MDA as a sponsor of the Hereditary Neuropathy Foundation, who will be hosting the Externally-led Patient-Focused Drug Development Meeting (PFDD).
Regenacy Pharmaceuticals Announces Collaboration with the CMTA to Advance Ricolinostat for the Treatment of Hereditary Neuropathy
Regenacy today announced a collaboration with the CMTA 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.
CMTA Announces Strategic Partnership with Acceleron to Advance CMT Treatment Options
"We are excited to establish this partnership with Acceleron as they work to advance their lead neuromuscular therapeutic candidate, ACE-083, through an ongoing Phase 2 trial in patients with CMT," said CMTA's CEO Amy Gray.
Toxic Medications List For Patients with Charcot-Marie-Tooth Disease
What is a Neurotoxic Medication? Neurotoxicity is the capacity of
