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

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