Tracey Sikora has studied rare diseases and repurposed drugs for more than a decade. Early career pre-clinical work investigating pentosan polysulfate for use in MPS I and cyclodextrin for the treatment of Niemann-Pick Type C led to further investigation in clinical trials. Later, she led clinical trials for Familial Hypercholesterolemia, Fabry disease, Castleman disease, and others by working alongside partners at biotech companies and rare disease organizations. Critical to clinical trial success is developing study protocols that adapt to the challenging realities of conducting clinical research in the real-world.