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Idebenone treatment in Leber's hereditary optic neuropathy: rationale and efficacy
Leber's hereditary optic neuropathy (LHON) is a rare mitochondrial retinopathy, caused by mutations in subunits of complex I of the respiratory chain, which leads to elevated levels of oxidative stress and an insufficient energy supply. This molecular pathology is thought to be responsible for the dysfunction and eventual apoptotic loss of retinal ganglion cells in the eye, which ultimately results in blindness. Many strategies, ranging from neuroprotectants, antioxidants, anti-apoptotic- and anti-inflammatory compounds have been tested with mixed results. Currently, the most promising compounds are shortchain quinones that have been shown to protect the vision of LHON patients during the early stages of the disease. This commentary gives a brief overview on the current status of tested therapeutics and also addresses future developments such as the use of gene therapy that hopefully will provide safe and efficient therapy options for all LHON patients.
History
Publication title
Expert opinion on orphan drugsIssue
4Pagination
130-135ISSN
2167-8707Department/School
School of Pharmacy and PharmacologyPublisher
Informa HealthcarePlace of publication
UKRepository Status
- Restricted