The CMTRF has joined the European Charcot-Marie-Tooth Federation (ECMTF), a voluntary non-profit federation based in Belgium, created to unite advocacy groups that raise awareness, fund research, and promote better care for people with CMT. Among the primary goals of the ECMTF are funding research projects that are beyond the scope of the individual organizations and promoting communication and collaboration between researchers, specialists, clinicians, and CMT organizations. This aligns closely with CMTRF’s mission to raise funds to invest in science that will lead to treatments and cures for CMT And introduce CMT to the greater drug development community, something that has rapidly increased the number of experts working to solve the disease.
Important Research News Announcements
- Combined RNA interference and gene replacement therapy targeting MFN2 as proof of principle for the treatment of Charcot-Marie-Tooth type 2A November 25, 2023
- Mouse models of human CNTNAP1-associated congenital hypomyelinating neuropathy and genetic restoration of murine neurological deficits October 19, 2023
- Intravenous Administration of an AAV9 Vector Ubiquitously Expressing C1orf194 Gene Improved CMT-Like Neuropathy in C1orf194-/- Mice October 16, 2023
- Clinical trials in Charcot-Marie-Tooth disorders: a retrospective and preclinical assessment September 22, 2023
- Structures of wild-type and selected CMT1X mutant connexin 32 gap junction channels and hemichannels August 30, 2023
- AAV9-mediated SH3TC2 gene replacement therapy targeted to Schwann cells for the treatment of CMT4C August 28, 2023
- Proteostasis plays an important role in demyelinating Charcot Marie Tooth disease August 19, 2023
- Gene replacement therapy in two Golgi-retained CMT1X mutants before and after the onset of demyelinating neuropathy August 2, 2023
- Amlexanox: Readthrough Induction and Nonsense-Mediated mRNA Decay Inhibition in a Charcot-Marie-Tooth Model of hiPSCs-Derived Neuronal Cells Harboring a Nonsense Mutation in GDAP1 Gene July 24, 2023
- Monoallelic variation in DHX9, the gene encoding the DExH-box helicase DHX9, underlies neurodevelopment disorders and Charcot-Marie-Tooth disease July 18, 2023
