Andrew Marella Receives Cyclodextrin Infusion - October 2015

Each month since December 2013, Andrew has received a lumbar puncture infusion of Cyclodextrin as part of a Phase 1 clinical trial at the National Institutes of Health. It is our great hope that Cyclodextrin is the most beneficial medicine yet for treating Niemann-Pick type C disease, improving both the patients' quality of life and their longevity; both critical to our efforts to find a real cure for all of these children. No drug has been shown to be as effective in NPC animal models as Cyclodextrin. The clinical trial is now moving forward into an FDA and EMA approved Phase 2/3 trial, lead by pharmaceutical company Vtesse, hoping to adequately demonstrate Cyclodextrin's effectiveness.