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CMTA-STAR Partner Applied Therapeutics Presents 24-Month CMT-SORD Data at PNS 2025

CMTA-STAR Alliance Partner Applied Therapeutics presented 24-month Phase III data on govorestat for CMT-SORD at PNS 2025, showing sustained clinical benefit, MRI-based disease slowing, and continued safety. These findings support the potential for the first approved treatment for CMT-SORD and mark progress toward an expected NDA submission.

CMT-SORD: Bringing the Science Down to Earth | Shooting Star Ep. 3

Shooting Star makes CMT science easy to understand! In this episode, the CMTA youth group explores CMT-SORD, a rare form of CMT linked to mutations in the SORD gene. Learn how it’s diagnosed, what makes it unique, and why research is gaining momentum. Watch now and stay informed!

Alliance Partner NMD Pharma A/S Launches Phase II SYNAPSE-CMT Trial with First Patient Dosed at CMTA Center of Excellence

CMTA-STAR Alliance Partner NMD Pharma A/S has launched the SYNAPSE-CMT Phase II trial, with the first patient dosed at a CMTA Center of Excellence. This study is evaluating NMD Pharma's NMD670, an investigational therapy aimed at improving nerve-to-muscle communication in adults with CMT1 and CMT2. The trial is now enrolling at sites in the U.S. and Europe. Learn more about how CMTA supports this critical research and how you can get involved.