New Data from the Open-Label PLEO-CMT-FU Trial Shows Sustained Benefit with PXT3003 in Patients with Charcot-Marie-Tooth Disease Type 1A After 5 Years of Total Trial Time
The entire CMT community is hopeful that PXT3003 could be the first approved therapy for this debilitating disease.
- The entire CMT community is hopeful that PXT3003 could be the first approved therapy for this debilitating disease.
- All randomized CMT1A patients who completed the PLEO-CMT trial (treated with PXT3003 or placebo) were eligible to pursue treatment with PXT3003 in the PLEO-CMT-FU trial.
- This trial enrolled a total of 187 patients and was designed to primarily assess the long-term safety and tolerability of PXT3003.
- Patients treated with placebo in the PLEO-CMT trial were randomized in PLEO-CMT-FU to receive LD or HD of PXT3003.