What to Expect as a Participant in a Gene Therapy Clinical Trial
All times below listed in ET
Planned by the Patient Outreach Committee. Held in collaboration with the National Organization for Rare Disorders (NORD).
Cochairs: Emily McGinnis and Kimberly Goodspeed, MD
Register for What to Expect as a Participant in a Gene Therapy Clinical Trial
If you're a patient, caregiver, volunteer advocate, or an employee of a non-profit patient advocacy organization, learn how to receive free registration for this workshop.
Session 1 – The Process: From Setting Up a Clinical Trial to Being a Participant
1:00 – 3:00 p.m.
Current Trends in Clinical Trials
Kimberly Goodspeed, MD, UT Southwestern Medical Center
How a Clinical Trial is Set Up and Run
Megan Waldrop, MD, Nationwide Children’s Hospital
Complexities of Eligibility: Why Do We Have to Exclude Patients?
Heather Lau, MD, Yale University School of Medicine, Ultragenyx Pharmaceutical Inc.
What to Expect Before and During a Clinical Trial
Nicolas Abreu, MD, NYU Langone Health
What to Expect After a Clinical Trial
Perry Shieh, MD, PhD, FAAN, University of California, Los Angeles
Panel Q&A moderated by Kimberly Goodspeed, MD, UT Southwestern Medical Center
Break: 3:00 – 3:30 pm
Session 2 – Risks, Challenges, and Hopes
3:30 – 5:00 p.m.
To Participate or Not To Participate: A Discussion of the Risks, Expectations, and Considerations for Participation in a Gene Therapy Clinical Trial
Diana Bharucha-Goebel, MD, National Institutes of Health
Participant Challenges and Making the Decision
- Emily McGinnis, Taysha Gene Therapies
- Pat Furlong, Parent Project Muscular Dystrophy
- Natalie Wheatley
- Julia Vitarello, Mila’s Miracle Foundation
Panel Q&A moderated by Emily McGinnis, Taysha Gene Therapies