Heather Lau

Company: Ultragenyx Pharmaceutical Inc

Job title: Executive Director

Bio:

Dr. Lau is executive director of Global Clinical Development at Ultragenyx and is a board-certified neurologist with special qualifications in pediatrics. Prior to joining industry in 2021, Dr Lau was the Director of Lysosomal disorders and head of the Division of Neurogenetics at NYU Langone, where she ran a clinic to diagnose and manage adult and pediatric patients with a variety of rare genetic disorders, as well as conducted over 20 clinical trials in rare genetic diseases that were investigating small molecule, protein-based and gene therapies.

Seminars:

Panel Discussion: Unpicking the Complexities of External Control Arms 12:00 pm

Identifying the challenges associated with conducting robust external control arm studies in small, heterogenous patient cohort Methodological considerations for using external control arms to credibly demonstrate the effectiveness of a rare gene therapy Key lessons learned from clinical trial designs incorporating external controls Considerations for using natural history studies as external control armsRead more

day: Clinical - AM

NAVIGATING THE COMPLEXITIES OF RARE GENE THERAPY CLINICAL TRIALS 9:00 am

Understanding the nuances of initiating and running clinical trials for rare patient populations Who are the key stakeholders a clinical team should be collaborating with when commencing a trial? Collaborating effectively with patient groups to optimize the design and conduct of trials Improving the accuracy of finding patients in rare disease populations Monitoring, mitigating, and…Read more

day: Workshop Day - track b