A definitive treatment for hereditary optic neuropathies is missing; nevertheless, some options are currently available and are under continuous evolution. They range from drugs designed to overcome the metabolic defect and compensate the mitochondrial dysfunction, to gene therapies and stem-cell-based approaches.

Idebenone, a coenzyme Q10 analogue, is the most studied molecule with antioxidant properties that has shown to improve visual acuity in Leber’s Hereditary Optic Neuropathy (LHON), and hopefully it will be offered soon in other mitochondrial optic neuropathies.

The ultimate goal for the treatment of hereditary optic neuropathies is the correction of the genetic defect, or the prevention of its transmission to progeny. The delivery of the wild-type gene in LHON patients’ eyes has shown some positive results in terms of visual outcome, but  further studies are needed.