The CMT Research Foundation has funded a new study at University of California San Diego led by Dr. Uri Manor to build an end-to-end pipeline for finding and validating a genetic treatment for CMT2A. CMT2A is a progressive disease caused by mutations in a gene...
The CMT Research Foundation has funded a new study at Johns Hopkins University to find a blood-based biomarker that could measure whether future CMT treatments are working. The research, targeted at subtype CMT2C, is led by Dr. Jeremy Sullivan and Dr. Charlotte...
The CMT Research Foundation has invested in a research project with Dr. Wolfgang Pernice at Columbia University to develop a novel tool for clinical endpoint monitoring of Charcot-Marie-Tooth (CMT) disease progression entitled DANCER: Digital Assessment of Natural...
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,”...
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...
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...
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...
CMT Research Foundation has invested in a research project with Asha Therapeutics to investigate the therapeutic potential of inhibiting SARM1 (Sterile alpha and TIR motif-containing protein 1) using their novel drug, ASHA-624, for the treatment of Charcot-Marie-Tooth...
Researchers at Vanderbilt University have made significant progress in the development of small molecules aimed at treating Charcot-Marie-Tooth disease types caused by mutations in the PMP22 gene, which include CMT1A, CMT1E and hereditary neuropathy with liability to...
CMT4B1 is a severe form of Charcot-Marie-Tooth disease caused by mutations in the gene that makes Myotubularin-related protein 2 (MTMR2). Dr. Alessandra Bolino, PhD, discovered that mutations in this gene affect the production of fats, called phospholipids. Among...