Natural History Study

The clinical development team at Med-Life Discoveries together with the team at Nemours Children’s Hospital spent over a year finalizing the design of the Natural History Study. Unlike a registry, a Natural History Study is designed just like a clinical trial, just without the inclusion of a therapeutic intervention. As such, the Natural History Study has inclusion and exclusion criteria, a defined list of assessments that is completed on each patient, and a schedule for repeating these assessments (every 6 months). All study assessments are standardized, allowing for comparison of the data between patients, as well as within a single patient over time. Participants in the Natural History Study will to undergo a variety of clinical assessments, both at Nemours Children’s Hospital, as well as surveys completed at home. If you are interested in participating in the study more information on the criteria, assessments and clinical contacts is available here.