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Danon disease is a rare, multisystem X-linked genetic disorder caused by variants in the LAMP2 gene. In children and young adults, it is defined overwhelmingly by its cardiac effects . What is far less appreciated is that the eye is frequently involved and that the retinal findings can appear before the systemic diagnosis is established.

Rare retinal diseases initially were the primary targets of gene therapy, but that is no longer the case. The focus has expanded beyond those to also include more common, chronic diseases such as diabetic macular edema and wet age-related macular degeneration. During the first half of 2026, that trend was evident.