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2015-12: Compassionate treatment with idebenone of recent onset Leber Hereditary Optic Neuropathy

Children and inhertable diseases
Drug research
Investigator Initiated Research
Inclusion open since 2-10-2015

Leber Hereditary Optic Neuropathy (LHON) is caused by a mutation of mitochondrial DNA which impairs the Complex I (electron transport chain) function. LHON usually presents as rapid loss of central vision in one eye, soon followed by the fellow eye. Retinal ganglion cells, in particular, are affected. Onset typically between 15 - 35 years of age with visual acuity (VA) progresses to less than 0.1. It has been suggested that idebenone may help to improve mitochondrial function.

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