Rare Disease Day - Clinical Gene Therapy: The Future is Now

Rare Disease Day

Clinical Gene Therapy: The Future is Now

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. 

RSVP and Abstract Submission

Research Poster Abstract Submission

Research poster abstract submissions will be accepted on basic, translational, and clinical research related to rare diseases and their treatments.

Co-Sponsors of Rare Disease Day

Center for Orphan Drug Research
University of Minnesota Medical School Stem Cell Institute


James M. WilsonJames M. Wilson, MD, PhD

Rose H. Weiss Professor and Director
Orphan Disease Center, University of Pennsylvania


Patient Perspectives on Gene Therapy - Patients and caregivers representing several rare disease communities share their thoughts, hopes and concerns regarding gene therapy

Rare Disease Day


Friday, March 1st, 2019
11:00am to 2:30pm


Graduate Hotel, Minneapolis, MN


Patient Advocacy Group Breakfast

Poster Session
Research posters related to rare diseases or orphan drug research from investigators and their trainees

11:45 Lunch

12:00-2:00 Program

2:00-2:30 Networking