Experts InSight Updates in the Treatment of Leber Hereditary Optic Neuropathy (LHON)
Apr 17, 2025
Dr. Nancy Newman, a leading ophthalmology and neurology expert at Emory University, discusses groundbreaking advances in treating Leber hereditary optic neuropathy (LHON). She highlights the promise of idebenone and the intricacies of gene therapy targeting specific mutations, particularly the beneficial effects seen with the 11778 mutation. The conversation delves into clinical trial outcomes, challenges in medication accessibility, and the importance of community support for patient recruitment. New insights could pave the way for improved patient outcomes and understanding of this rare condition.
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Idebenone's Dual Mechanism
- Idebenone is a short-chain benzoquinone that stimulates ATP production and acts as a potent antioxidant.
- It bypasses complex one of mitochondrial respiratory chain, targeting the common mutations in LHON.
Idebenone Dosage and Cost
- Use idebenone at 300 mg orally three times daily with meals for adult LHON patients.
- Inform patients it's safe but expensive, costing about $2,000 per year out of pocket.
Idebenone's Variable Efficacy
- Idebenone stabilizes vision in subacute phase and promotes vision recovery in chronic phase of LHON.
- Its effectiveness varies by mutation, with most benefit seen in 11778 mutation patients over 18 years old.