Penn Orphan Disease Center

The Orphan Disease Center (ODC) of the University of Pennsylvania (Penn) is led by Dr. Jim Wilson. The ODC was formed to facilitate and fund research and to develop transformative therapies for rare diseases with significant unmet need.

As part of our research collaboration with Penn's Gene Therapy Program, we have access to the ODC's insights and capabilities in the study of rare diseases. We leverage our close working relationship with the ODC to develop historical and prospective external data for each disease for use in building comparable patient profiles of participants in interventional trials. In addition, we believe the ODC's close ties to leading clinical centers for rare, monogenic central nervous system disorders will improve our ability to identify potential patients for trial enrollment, and enhance patient retention and data quality. The ODC is currently performing a natural history study of patients with GM1 funded by us.

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