Critical need for harmonized post marketing surveillance in gene therapy | Virtual Summit 2020
With regulatory approval of the first gene therapy product expected, there is an urgent need for a mechanism to collect long-term data on safety and variability and durability of efficacy. There will be elements required by regulators for post marketing surveillance and additional data needed to enhance our understanding of gene therapy outcomes and their […]
The Challenge of Evidence Generation
In the Tuesday morning WFH session on global surveillance and collaboration, Glenn Pierce, explores the challenges of evidence generation.
The "My Life, Our Future" Hemophilia Genetic Analysis Project
In the Wednesday morning lab sciences session on the genetic laboratory for bleeding disorders, Barbara A. Konkle discusses the work and experiences, to date, of the My Life, Our Future hemophilia genetic analysis project.