Understanding Gene Therapy: From Fundamentals to NGGT’s Clinical Trial

Gene therapy is one of the most talked-about areas in rare disease research, but it can also be hard to understand. This informative webinar that breaks down the basics of gene therapy and explores how it’s being studied for PKU.

Dr. Jerry Vockley explains how gene therapy works, shares an overview of current clinical techniques and discusses important safety considerations. NGGT will then share about their ongoing gene therapy clinical trial for PKU and what it could mean for the future of treatment.

To learn more about the NGGT trial, see their study flyer: https://www.npkua.org/wp-content/uplo...

This study was approved by the NPKUA Registry Committee for information to be shared with participants in the PKU Patient Registry. To see more approved studies, visit our website: https://www.npkua.org/research/pkustu...

This content is being shared for informational purposes only to highlight active PKU studies that have been reviewed by the NPKUA Registry Committee. NPKUA does not provide endorsement nor recommendation to participate in any particular study or trial. If you are interested in participating in a particular study, please review the details of the study or consult with your medical team if needed.