Leber Hereditary Optic Neuropathy

Leber hereditary optic neuropathy (LHON) is a rare disease that leads to sudden vision loss, most commonly in adolescence and early adulthood. There is no cure for LHON, and there are no therapeutics available to reliably prevent, halt, or reverse the onset or progression of LHON vision loss. Most patients with LHON rapidly reach and surpass the legal blindness threshold and must quickly re-learn how to navigate a world in which they can no longer recognize faces, drive, or read—a process that incurs significant social, emotional, and financial burdens.   

LHON research has been constrained by limited, inconsistent funding, which hinders progress from early discovery to the development of treatments. Every investment in LHON research has implications not only for patients with LHON but also for patients with other conditions that share mechanisms or cell types with LHON, including mitochondrial diseases, optic neuropathies, and neurodegenerative disorders.  

Milken Institute SPARC is partnering with LHON Collective to identify areas where philanthropic investment in research and development can have the greatest impact on transforming the LHON research and therapeutic landscapes.