The Penn Medicine Orphan Disease Center (ODC) was established to promote the development of therapies across a broad range of orphan diseases.

Each of these diseases by definition is rare but collectively represents a substantial health care burden. Most orphan diseases manifest in children with premature mortality and/or significant disability and little is available in terms of treatments. In order to assemble a critical mass of intellectual and patient resources for any one disease, the ODC will reach beyond the borders of Penn and promote inter‐institutional collaborations/partnerships. The ODC will develop transformative therapies using platform technologies that can be deployed across multiple rare diseases. We will emphasize disorders with substantial unmet need independent of their incidence and will strive to assure access to patients of all populations.