MD, Mayo Medical School
Bone tumors, clotting disorders in children
NCT02534844: "A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled 3-Part Trial of 2-hydroxypropyl-_-cyclodextrin (VTS-270) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease", Co Investigator