CMTA has partnered with Shift Pharmaceuticals to evaluate an antisense oligonucleotide (ASO) for the treatment of CMT1A. The collaboration will use CMTA-STAR preclinical testing capabilities to assess the therapeutic potential of an ASO designed to reduce PMP22 overexpression and support advancement toward clinical trials.
CMT2F Biomarkers and Outcome Measures
With CMTA support of $302,071, an international team of researchers from the Inherited Neuropathy Consortium...
Schwann Cell-Targeted Gene Therapy Approaches to Treat CMT1A and Other Demyelinating Neuropathies
With CMTA support of over $160,000, an international team of researchers is developing genetic therapy...
Applied Therapeutics – INSPIRE
This study is designed to assess the efficacy and safety of AT-007 treatment in patients...
CMTA Seed Money Draws NIH Support for Type 2 Gene Editing, Neurofilament Projects
CMTA seed funding has helped secure additional NIH support for two CMT type 2 research projects focused on gene editing and neurofilament restoration. The NIH awards build on CMTA-funded pilot work and reflect the role of early philanthropic investment in advancing translational CMT research toward therapeutic development.
CMTA | STAR Awards $354,826 For Charcot-Marie-Tooth Clinical Trial
CMTA-STAR has awarded $354,826 to support a clinical trial evaluating L-serine in patients with Hereditary Sensory Neuropathy 1, a rare form of CMT. Led by Dr. Mary Reilly at University College London, the study will assess disease progression using MRI muscle fat fraction as a primary outcome measure, advancing clinical trial readiness for inherited neuropathies.
Development of a new CMT1A research toolbox: an alternative human stem cell-based strategy
With CMTA support of $98,890, researchers at Hasselt University in Belgium, led by Esther Wolfs,...
2A Biomarkers and Outcome Measures
With CMTA support of $572,055, an international team of researchers from the Inherited Neuropathy Consortium,...
CMTX1 Biomarkers and Outcome Measures
With CMTA support of $529,971, an international team of researchers from the Inherited Neuropathy Consortium,...
1B Biomarkers and Outcome Measures
With CMTA support of $529,971, an international team of researchers from the Inherited Neuropathy Consortium,...
