Pharnex PXT3003 study.
Pharnext have confirmed their latest study. The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease – Type 1A. This double-blind study will assess in parallel groups 1 dose of PXT3003 compared to Placebo in CMT1A patients treated for 15 months. The study will be conducted in approximately 48 sites worldwide. No UK centres have engaged with this study. We will continue to bring the results of these studies as and when they are available.