Phase 3 TransportNPC study to continue based on the independent DMC review of safety and efficacy data at prespecified 48-week interim analysis
Data on the investigational candidate Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin), indicates that it is well-tolerated and has a safety profile consistent with the previously completed phase 1 and 2 studies and ongoing phase 1 open-label extension study
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