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Diana Bharucha-Goebel, MD Joins the CMTA-STAR Advisory Board
…is also interested in better understanding how we efficiently and safely target the peripheral nervous system with viral mediated gene transfer treatment approaches, studying total vector genome dose, parallel preventative…
Natural History of TRPV4 Neuromuscular Disease – CMT2C
…in animal models of this condition has shown that disease mutations cause TRPV4 to be overactive, and drugs that block the channel are effective as treatments in these models. This…
2021 STAR Accomplishments
…the body responds to a treatment for a disease) Initiatives in CMT1A, CMT1X, CMT2A, CMT1B and others in process: INC progress in developing muscle MRI and blood sample measurements of…
CMTA’s $240K Investment Targets Gene Replacement Therapy in CMT4 Subtypes
The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic funder of Charcot-Marie-Tooth (CMT) disease research aimed at bringing treatments to patients, announced today an investment of $240K into a new gene replacement…
How to Decide If Foot Surgery Is Right for You
NMD Pharma Receives FDA Approval to Initiate Phase II Clinical Trial of NMD670 in CMT Patients
…clinical trial for their investigational drug, NMD670, in patients with Charcot-Marie-Tooth disease (CMT). This trial, named SYNAPSE-CMT, marks a significant milestone in the journey towards finding effective treatments for CMT….
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…
Hell’s Kitchen Broadway Show
09/28/2024 @ 2:00 pm – 5:00 pm – Hell’s Kitchen is a new musical created by 15-time Grammy Award winner Alicia Keys. Featuring her new original songs and iconic anthems in a story inspired by her own New York experience, this is the kind of Broadway musical of which dreams are made. Hell’s Kitchen has been nominated for thirteen Tony Awards including Best […]
CMTA Centers of Excellence
CLINICAL EXPERTISE, COMPREHENSIVE EVALUATION AND CARE, INFORMATION CRUCIAL FOR CLINICAL TRIALS CMTA’s community-led, community-driven mission is to support the development of new treatments for Charcot-Marie-Tooth disease (CMT), to improve the…
Gilles Bouchard Takes Helm of CMTA Board
…that while he is “excited and delighted” with the progress made by the CMTA’s STAR research program, there’s still much work to be done before a first drug treatment for…