Thesis defense Patty Dhooge-Verberne (Donders series 580)

19 January 2023

Promotors: prof. dr. C. Hoyng, prof. dr. B. Klevering

Treating Stargardt Disease

Stargardt disease is an inherited retinal disorder that can lead to blindness. To date, there is no commercially available treatment. Currently, 23 different therapeutic agents are being developed of which thirteen are being tried in 24 registered clinical trials. With this, commercially available treatments for Stargardt disease are just around the corner. This thesis comments on the expected treatment options and addresses several challenges regarding clinical trials for Stargardt disease. This thesis aims to provide guidelines for future clinical trials for STGD1 concerning selection of the right cohort, endpoint(s), timing and trial design. This thesis also describes the Stargardt Remofuscin Treatment Trial in which patients are actually treated for Stargardt disease in the context of research. The findings presented in this thesis make an contribution to the prevention of blindness in patients with Stargardt disease.