- 2023 CMTA-STAR Celebrating our Collective AchievementsSignificant Research Investment Made in 2023: We are excited to share that in 2023, the CMTA has ongoing and committed investments of $5,000,00 in research dedicated to finding effective treatments and ultimately a cure for Charcot-Marie-Tooth disease. Support for Natural History Studies Drives ...Read more ...
- ToolGen CMT1A Treatment Receives Orphan Drug DesignationCMTA Alliance Partner ToolGen announced December 18, 2023 that it received Orphan Drug Designation (ODD) for its rare disease gene correction treatment, TGT-001, from the U.S. Food and Drug Administration (FDA). TGT-001 targets Charcot-Marie-Tooth (CMT), a rare genetic disease related ...Read more ...
- Pharnext Announces Disappointing Results for CMT1A Drug Trial PXT-3003Published: December 11, 2023 French biopharmaceutical company Pharnext SA announced Dec. 10 that the initial results from its Phase III clinical trial (PREMIER trial) for CMT1A did not meet expectations. The company said in a news release that analyzing the results was ...Read more ...
- Novartis Secures $1.3B Deal for CMT Treatment CKD-510Novartis is making significant strides in the field of Charcot-Marie-Tooth (CMT) disease treatments, having recently entered into a substantial $1.3 billion deal with Chong Kun Dang Pharmaceutical Corp (CKD). The South Korean pharmaceutical company announced on November 6th that it ...Read more ...
- Therapeutic Inactivation of CMT2 Disease Alleles with CRISPRNew technologies in gene editing such as CRISPR/Cas9 present a promising opportunity for the treatment of genetic diseases like CMT at the root cause – often just a single base pair or “letter” change in the DNA sequence. Research lead ...Read more ...