ABCA4-associated disease in childhood and adolescence- a phenotype study.

Abstract

Key messages: WHAT IS KNOWN : So far, observational studies have primarily examined adults and have used these findings to define endpoints for interventional studies, which also serve as a reference for interventional studies in children  Most clinical trials currently use the measurement of definitely decreased autofluorescence WHAT IS NEW : We have closely examined the variability of Stargardt disease in children  Our results suggest that the same inclusion criteria and endpoints used for adults should not be applied to children, e.g. definitely decreased autofluorescence is not suitable in pediatric cohorts because it has only a very low prevalence.