PXT3003 Shows Sustained Safety, Benefits for Patients With CMT1A

PXT3003 Shows Sustained Safety, Benefits for Patients With CMT1A

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PXT3003 Shows Sustained Safety, Benefits for Patients With CMT1A

High-dose PXT3003, an investigational therapy for Charcot-Marie-Tooth disease type 1A (CMT1A), continues to show long-term safety and efficacy, according to an early analysis of a Phase 3 extension study. All participants showed improvements on the Overall Neuropathy Limitation Scale (ONLS), a measure of physical disability. “These new results from the interim analysis of the ongoing open-label Phase 3 extension study show very promising safety and efficacy data of PXT3003 in CMT1A after more than four…

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