Stargardt Disease (STGD1) is a rare inherited retinal disease (1 in 8,000 - 10,000 individuals) that afflicts approximately 30,000 individuals in the US. It is an autosomal recessive disease most commonly due to ABCA4 gene mutations, and is a devastating neurodegenerative disease involving rapid growth of lesions (retinal geographic atrophy, or GA) in the central part of the retina known as the macula. 

Early onset of the disease is highly aggressive and occurs in childhood/adolescence (7-18 years of age) with a subsequent peak in early adulthood, often correlating with a worse prognosis. Late onset is generally associated with a milder disease course. The disease involves a rapid decline in visual acuity (significantly impaired best-corrected visual acuity (BCVA)), bilateral central vision loss, blurred vision, and central scotomas. 

The pathophysiology of the disease is primarily driven by excessive accumulation of retinaldehydes and retinal bisretinoids. Inflammation in the retina via complement system dysregulation is also a major contributing factor in disease progression. Currently, there are no therapies available for STDG1 patients.