Leber's hereditary optic neuropathy

Leber’s hereditary optic neuropathy

Leber’s hereditary optic neuropathy (LHON) is a severe mitochondrial optic neuropathy disease leading to central vision loss in both eyes.1–5 People living with LHON will often find it impossible to read, drive or recognize faces.

LHON is a rare eye disease that usually affects young, otherwise healthy individuals and is more common in men than women.2 LHON occurs in approximately 2 in every 100,000 people in Europe.1,2,6

Leber’s hereditary optic neuropathy

Impaired vision such as weak vision in one eye, discordant vision, color vision loss, severe loss of central vision and legal blindness occur in the majority of patients within 1 year of their symptoms becoming apparent.1,4,5 The visual defect in one eye is sudden, abrupt, painless and profound. This is typically followed by loss of vision in the other eye 1–3 months later.5

Normal Vision without impaired Vision
Normal Vision without impaired Vision
Severe Vision Loss caused by LHON
Severe Vision Loss caused by LHON

Causes and treatment

LHON is caused by mutations in genes in the mitochondria (small structures within cells that produce the energy the cells need to function). When such mutations are present, nerve cells in the retina at the back of the eye do not have enough energy to work properly (retinal ganglion cell dysfunction), leading to the characteristic loss of vision.4,5

For a short animation about LHON, please click on the video below.

As with other diseases, early diagnosis and treatment are important to try to preserve eyesight respectively to improve vision loss and by vision stabilization.5

If treated early – even after blindness has occurred – appropriate treatment of vision loss can lead to vision recovery.7

Causes, Symptoms and Treatment of LHON

Image adapted from: Gueven N. Biol Med 2014; 6(S3):1–6 and Howell N. Vision Res 1998; 38:1495–1504.

If you are affected by LHON in any way, or know of somebody who is, and would like to know more about the disease, please contact your local healthcare provider.

Santhera and LHON

Raxone – the first pharmaceutical product for a mitochondrial disorder – was approved in the European Union in September 2015 for the treatment of patients with LHON.8,9

If you are interested in learning more about Raxone in LHON, please click here

If you are a patient currently taking Raxone, please click here for additional information on how to take Raxone.

If you are a registered healthcare professional, please click here to find out more about Raxone, the first and only approved treatment for LHON.8,9



Mascialino B, et al. Eur J Ophthalmol 2012; 22:461–465.


OMIM. Leber Optic Atrophy. 2012. click here. Accessed September 2015.


EMA. Public summary of opinion on orphan designation. Idebenone for the treatment of LHON. 2011.


Yu-Wai-Man P, et al. Prog Retin Eye Res 2011; 30:81–114.


Sadun AA, et al. Expert Rev Ophthalmol 2012; 7:251–259.


EMA. Assessment report: Raxone (idebenone). 2015.


Howell N. Vision Res 1998; 38:1495–1504.


Raxone SmPC, September 2015.


EMA. EPAR summary for the public: Raxone (idebenone). 2015.