Charcot Marie Tooth 1A

Published on January 7th, 2020

Pharnext Announces Encouraging Data from Open-Label Phase 3 Extension Study of PXT3003 in Charcot-Marie-Tooth Disease Type 1A (CMT1A) 

  • Results suggest sustained safety and efficacy of PXT3003 in CMT1A patients after 25 months of total trial time (Phase 3 trial + open-label extension study)
  • CMT1A patients showed improvement or stabilization of disease as measured by the Overall Neuropathy Limitations Scale during the open-label Phase 3 extension study

The Press Release is available at this link:

To read latest news