News & Stories
See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.
Lighting the Future: A Mother’s Fight for a Cure
The Greatest Gift: Kaileen Nelson, Canada, Living with CMT2D Every December, Kaileen looks for light. Not just the glittering bulbs strung across her Canadian neighborhood — but the kind of light that reminds her that hope still exists. Because for Kaileen, simple...
CMT Research Foundation to Benefit from a Portion of Ticket Sales from Alan Jackson’s Last Call: One More for the Road – The “Finale”
Alan Jackson is ready to serve up one more round before he retires from touring—and he’s inviting a few friends to join him! Last Call: One More for the Road - The Finale – the last full-length concert of Jackson’s touring career – will take place Saturday, June 27,...
CMT Research Foundation Funds Elpida Therapeutics to Launch Manufacturing of a Gene Therapy Drug for CMT4J
CMT Research Foundation announced they have funded Elpida Therapeutics to launch manufacturing of a gene therapy drug for Charcot-Marie-Tooth disease type 4J. The announcement came at the 2025 Global CMT Research Convention. “Our ELP-02 gene therapy is trial-ready,”...
Dr. Meghan Drummond Joins the CMT Research Foundation as Vice President of Research and Drug Development
Dr. Meghan Drummond, PhD, has joined the CMT Research Foundation as vice president of research and drug development. She will lead the Foundation’s research and drug development strategy, overseeing the advancement of promising therapeutic approaches for...
CMT Research Foundation Invests in the Next Generation of CMT Researchers
The CMT Research Foundation has awarded funding to four new research projects through its Emerging Researcher Award program, which provides small, high-impact grants to promising scientists in academia and industry. Spanning multiple Charcot-Marie-Tooth disease...
Applied Therapeutics to Meet With FDA on New Drug Application for the Treatment of CMT-SORD
Applied Therapeutics, who will be speaking at the 2025 Global CMT Research Convention, recently announced that they will be meeting with the U.S. Food and Drug Administration, during the third quarter of 2025, to discuss a potential New Drug Application for...
Dr. Charles Abrams Discovers That Inosine Treatment Leads to Benefits in a CMT1X Animal Model
Dr. Charles Abrams, Professor of Neurology and Rehabilitation at the University of Illinois, Chicago, has discovered that inosine could potentially reduce nerve inflammation and offer moderate functional benefits in preclinical animal models of Charcot-Marie-Tooth...
Dr. Yulia Grishchuk Joins CMTRF’s Scientific Advisory Board
CMT Research Foundation is pleased to announce that Dr. Yulia Grishchuk, Ph.D., has joined the Foundation’s Scientific Advisory Board. The SAB is composed of distinguished scientific and clinical experts specializing in Charcot-Marie-Tooth disease and drug...
CMT Research Foundation Invests in XtRNA Bio to Develop Gene Therapy for CMT2D
CMT Research Foundation has invested in a research project with XtRNA Bio aimed to develop a new viral gene therapy for Charcot-Marie-Tooth disease type 2D. How GARS1 Mutations Drive CMT2D CMT2D is caused by mutations in the GARS1 gene, which encodes glycyl-tRNA...
A Father Who Defies ‘Impossible’: Joao Cardoso’s Journey with CMT
Joao Cardoso, Brittany, France, Living with CMT Joao Cardoso never imagined that he would be riding through the town of Brittany, France, in a wheelchair, while his eight-year-old son, Edgar, pedals his bike alongside him. Because of Joao’s Charcot-Marie-Tooth...