Eltrombopag
Skysona (lenadogene nolparvovec) is a gene therapy indicated for the treatment of Leber hereditary optic neuropathy (LHON) in adults with a confirmed mitochondrial DNA mutation associated with the ND4 gene. This one-time intravitreal therapy works by delivering a functional copy of the ND4 gene to retinal ganglion cells using an adeno-associated virus vector, potentially helping to prevent vision loss. As a brand-name medication, it represents a specialized treatment approach for this rare inherited mitochondrial disorder that primarily affects the optic nerve.
Skysona is a gene therapy used to treat Leber hereditary optic neuropathy (LHON) in adults with specific ND4 gene mutations.
Key facts at a glance
- RxNorm
- 2612554
- Generic
- Skysona
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Skysona is indicated for the treatment of Leber hereditary optic neuropathy (LHON) in adults who have a confirmed mitochondrial DNA mutation associated with the ND4 gene. LHON is a rare inherited condition that causes sudden vision loss, typically affecting young adults.
Skysona is an adeno-associated virus (AAV) vector-based gene therapy that delivers a functional copy of the wild-type ND4 gene directly to retinal ganglion cells via intravitreal injection. This allows the cells to produce the normal NADH dehydrogenase protein, potentially slowing or preventing further vision loss.
Skysona is administered as a single intra-vitreal injection per eye by a specialized physician. As a gene therapy, it is not intended to reverse existing vision loss but may help preserve remaining vision. Common considerations include potential immune responses to the viral vector and the need for confirmed genetic testing to identify eligible patients with ND4 mutations.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed August 9, 2026. · Read our editorial policy