Rare disease patient, caregiver and advocate presentations from the FDA’s “Clinical Trials: The Patient Experience” workshop are now available on the agency’s website, providing valuable patient points-of-view on gene therapy trials.

The virtual workshop, held on April 13, provided patients with a platform to share their experiences finding and participating in gene therapy trials. The workshop was the third in the Center for Biologics Evaluation and Research (CBER) educational RegenMedEd series.

In addition to an overview of the FDA’s gene and cell therapy priorities and processes, patient presentations included the clinical trial journey of a life-long hemophilia patient who enrolled in a gene therapy trial and was the first participant in that trial to receive the experimental treatment, as well as Suzette James’ experience caring for two children with CLN2 Batten disease and serving on the Batten Disease Support and Research Association’s board of directors.

Access the workshop slides, transcript and recording here.