Stargardt Disease Research: From ABCA4 Genetics to Emerging Treatments

A 2026 review explains how biallelic ABCA4 variants disrupt retinoid transport in Stargardt disease, leading to lipofuscin buildup, oxidative stress, complement activation, and retinal degeneration. It also highlights emerging pharmacological strategies and the importance of early diagnosis and specialist referral as disease-modifying therapies are pursued.