Proof of Concept for Levodopa Treatment in Rescuing Retinal Morphology and Visual Function in Albinism (Video)

There is a paucity of treatments for oculocutaneous albinism (OCA), a condition characterised by pigment deficiency, abnormal retinal development, and significant visual disability. L-DOPA, a signalling molecule which is essential for normal retinal development, is deficient in OCA. Residual plasticity of the developing retina in young children with albinism has been demonstrated, suggesting a post-natal window for therapeutic rescue. In this study, we investigate if post-natal retinal morphology and visual function in OCA can be improved through oral Levodopa supplementation, if administered during the critical period of neuroplasticity.