This year's program will further our understanding of gene therapy by hearing from a clinician and a panel of patients and parents. The clinician will provide an overview of current therapies and therapies that are in the pipeline, whereas the panel will feature patients who are currently receiving gene therapy and parents of children who are potential recipients. All are welcome to this free event.
Research Poster Abstract Submission
Research poster abstract submissions will be accepted on basic, translational, and clinical research related to rare diseases and their treatments.