Search results for: treatment
CMTA-STAR Delivery Science Portfolio: Four Parallel Approaches to Therapeutic Delivery in CMT
CMTA is funding four research projects targeting a fundamental barrier to CMT treatment: getting therapies past the biological defenses protecting peripheral nerves. The $523,000 CMTA-STAR investment supports complementary delivery approaches, including gene editing, RNA medicine, and nanoparticles, across multiple CMT subtypes.
CMTA-STAR Alliance Partner NMD Pharma Shares New SYNAPSE-CMT Trial Data
NMD Pharma shared new data from its Phase 2a SYNAPSE-CMT clinical trial evaluating the investigational drug ignaseclant during a late-breaking presentation at the 2026 MDA Clinical & Scientific Conference.
CMTA-STAR Preclinical Toolbox: A Validated Human iPSC Motor Neuron Assay for CMT2A Now Available
CMTA has developed and validated a human iPSC-derived motor neuron assay for CMT2A in collaboration with ANANDA Devices and BrainXell. Now available through the CMTA-STAR Preclinical Toolbox, the platform enables parallel drug screening and direct visualization of mitochondrial mobility impairment in CMT2A patient-derived neurons.
EMPOWER: Measuring Neurological Disease via Mobile Phone
Current tools for monitoring CMT often require clinic consultations and struggle to detect meaningful short-term...
Vera Bril, MD, BSc, FRCPC
Vera Bril is a Professor of Medicine (Neurology) at the University of Toronto, Director of...
CMTA Board of Directors Welcomes New Chair and Two New Members
The Charcot-Marie-Tooth Association named Wendy Arnone as chair of its Board of Directors and appointed...
We Go Where the Science Goes: 2025 CMTA Research Highlights
By Katherine Forsey, PhD Each year, I’m reminded that progress in CMT research is built...
Why participate in a clinical trial?
By Sue Bruhn, PhD Medical progress doesn’t happen by accident. Every treatment we rely on...
The Last Mile: Delivering CMT Therapies to the Nerves in Need
Developing a treatment is only half the challenge. The other half is getting it to...
CMTA Invests $141,000 to Study Hearing and Balance to Develop Better Clinical Trial Outcome Measures in Charcot-Marie-Tooth Disease
CMTA is supporting a two-year research project examining hearing and balance signaling to strengthen outcome measures in future CMT clinical trials.