Hereditary Optic Nerve Disorder Affecting the Liver
In a significant development for those affected by Leber Hereditary Optic Neuropathy (LHON), a rare inherited disease that can cause sudden vision problems, the UK has approved the use of the drug idebenone (Raxone) to treat patients aged 12 and older. This marks the first licensed pharmaceutical treatment to target the underlying mitochondrial dysfunction in LHON, offering hope for improving or stabilizing vision in affected patients.
LHON commonly presents with painless, progressive blurring and loss of central vision, typically starting in one eye and rapidly affecting the other within weeks or months. This vision loss can lead to severe visual impairment or blindness, primarily affecting young men and boys in their teens or early adulthood.
The condition is caused by a genetic mutation in mitochondrial DNA that disrupts energy production in retinal cells. Risk factors for LHON include male sex, young age, smoking, and alcohol consumption, which can increase risk and worsen prognosis due to additional mitochondrial stress.
Historically, treatment options for LHON have been limited to supportive care, such as nutritional supplements, counseling, and lifestyle management. However, idebenone works by restoring mitochondrial energy production in affected eye cells, potentially improving vision or slowing disease progression. Clinical trials show that idebenone can improve visual acuity and reduce further vision loss. The treatment involves oral tablets taken multiple times daily.
In addition to idebenone, investigational gene therapies such as lenadogene nolparvovec are being studied, showing promise in improving visual outcomes, especially when administered early and in patients with better baseline retinal structure.
It's important to note that LHON passes down through families through genes, with only females able to pass it on to their children. People with LHON in the family should avoid smoking and excessive alcohol consumption to reduce the risk of mitochondrial damage that leads to vision loss.
While idebenone offers a significant advancement in treating LHON, it's crucial to remember that each case is unique. Individuals with LHON should consult with their healthcare providers to discuss the best treatment options for their specific circumstances.
References:
- Mayo Clinic
- Genetic and Rare Diseases Information Center
- NHS UK
- Lenadogene nolparvovec (VY-A45) for Leber Hereditary Optic Neuropathy (LHON) - ClinicalTrials.gov
- Raxone (idebenone) for Leber Hereditary Optic Neuropathy (LHON) - ClinicalTrials.gov
- The emergence of idebenone (Raxone) as a licensed treatment for Leber Hereditary Optic Neuropathy (LHON) marks a significant milestone in science, especially for medical-conditions related to eye-health and neurological-disorders.
- LHON, a hereditary disease, can lead to blindness, highlighting the importance of health-and-wellness, mental-health, and lifestyle management in managing this condition.
- Beyond idebenone, ongoing research into gene therapies like lenadogene nolparvovec aims to further improve vision outcomes for people with LHON, underlining the ongoing pursuit of new treatments in various medical-conditions and industries.
