Search results for: treatment
CMTA Adds Drug Development Expert Martine Garnier to STAR Advisory Board
CMTA appoints drug development expert Martine Garnier, PhD, EMBA, to its STAR Advisory Board, strengthening its translational and therapeutic development expertise. Garnier brings more than two decades of experience in drug discovery and clinical development across academia, biotech, and pharmaceutical organizations.
SENSE HSN1 Clinical Trial
With $354,826 in CMTA funding, the SENSE Trial is an HSN1 clinical trial investigating a potential treatment for hereditary sensory neuropathy type 1, with active participation now complete and final data analysis underway.
Skilled Nonprofit Fundraiser Craig Zeltsar Brings Expertise to CMTA Board of Directors
CMTA has welcomed Craig Zeltsar to its Board of Directors, bringing decades of nonprofit fundraising and membership experience. Zeltsar’s background in donor-centered strategy, combined with his personal connection to the CMT community, strengthens CMTA’s leadership as it advances research and patient support.
CMTA Kicks Off CMT Awareness Month 2022
CMTA kicks off CMT Awareness Month 2022 with a month-long series of educational and community-driven activities designed to increase understanding of CMT. The campaign highlights people living with CMT and encourages public participation through digital engagement and awareness efforts.
CMTA Welcomes Software Entrepreneur Jon Pastor to Board of Directors
CMTA has appointed software entrepreneur Jon Pastor to its Board of Directors, adding leadership experience in technology and operations. Pastor brings a strong business background and a personal connection to the CMT community, supporting CMTA’s research and patient-focused mission.
The Charcot-Marie-Tooth Association Earns a Perfect Rating From Charity Navigator
CMTA has earned a perfect score from Charity Navigator, receiving a four-star rating for financial health, accountability, and transparency. The recognition places CMTA among a small group of health-related nonprofits meeting Charity Navigator’s highest standards for responsible stewardship.
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.
