Fellow
Austin Johnson
Stanford Medical School
Mentor
Joyce Teng, MD, PhD
Stanford School of Medicine
Overview
Epidermolysis Bullosa (EB) is a devastating hereditary blistering disease divided into subtypes based on unique mutations and location of affected skin areas. While there have been major breakthroughs for severe dystrophic EB, there is currently no FDA approved treatment for EB simplex (EBS), the most common subtype. Several early-phase EBS clinical trials have failed to obtain approval, which is partially due to a lack of appropriate scoring instruments to measure changes in disease severity. While tools to measure EB severity exist, such measurements are not suitable for EBS, creating challenges in presenting data to the FDA that reflects patients’ clinical outcomes during studies. Our aim is to develop and validate a new instrument for EBS clinical research by modifying existing tools, incorporating clinical experience from recent studies, and create standardized photographic representations of different disease severities before sending test photographs to key EBS opinion leaders for refinement and reliability analysis.
Status
This project was funded through a 2022 PeDRA Research Fellowship Grant.