With joint funding of $299,992 from CMTA and the Muscular Dystrophy Association (MDA), researchers at...
Shift Pharmaceuticals, Charcot-Marie-Tooth Association Partner on ASO Treatment for CMT1A
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.
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...
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.
