
Rafael Holdings Announces Continuation of its Phase 3 Study for the Treatment of Niemann-Pick Disease Type C1 (NPC1) ...
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 …