The ABCA4 protein (then called a “rim protein”) was first identified in 1978 in the rims and
incisures of rod photoreceptors. The corresponding gene, ABCA4, was cloned in 1997, and …

Endpoint development trials are underway across the spectrum of retinal disease. New
validated endpoints are urgently required for the assessment of emerging gene therapies …

Importance Sensitive outcome measures for disease progression are needed for treatment
trials of Stargardt disease. Objective To estimate the progression rate of atrophic lesions in …

Abstract The Progression of Atrophy Secondary to Stargardt Disease (ProgStar) studies
were designed to measure the progression of Stargardt disease through the use of fundus …

Importance Functional end points for clinical trials investigating the efficacy of emerging
treatments for Stargardt disease type 1 (STGD1) are needed. Objective To assess the yearly …

Stargardt disease, the most common inherited macular dystrophy, is characterized by vision
loss due to central retinal atrophy. Although clinical trials for Stargardt are currently …

Purpose Mean sensitivity (MS) derived from a standard test grid using microperimetry is a
sensitive outcome measure in clinical trials investigating new treatments for degenerative …

Retinal microperimetry (MP) is a procedure that assesses the retinal sensitivity while the
fundus is directly observed, and an eye tracker system is active to compensate for …

The aim of this review article is to describe the specific features of Stargardt disease and
ABCA4 retinopathies (ABCA4R) using multimodal imaging and functional testing and to …

Purpose: To investigate the effect of disease progression on the monocular preferred retinal
locus (PRL) of the better eye (BE) and worse eye (WE) of patients with central vision loss …