Luxturna: Used to treat Leber congenital amaurosis (LCA) type 2.

Brief information about it:
Luxturna is a gene therapy medication used to treat Leber congenital amaurosis (LCA) type 2, a rare inherited eye disease that causes severe vision loss from birth. It is the first gene therapy approved by the U.S. Food and Drug Administration (FDA) for a genetic eye disease.
Uses:
Leber congenital amaurosis (LCA) type 2: Luxturna is specifically approved for the treatment of LCA type 2, a genetic condition that causes vision loss due to a mutation in the RPE65 gene.
Mechanism of action:
Luxturna contains a functional copy of the RPE65 gene. When injected into the eye, it replaces the defective gene in retinal cells, allowing them to produce a protein necessary for vision.
Elimination half life:
The elimination half-life of Luxturna is not applicable, as it is a gene therapy medication and not a traditional drug. Once injected, the viral vector carrying the RPE65 gene becomes part of the patient’s cells and is not eliminated from the body.
Route of Administration:
Luxturna is administered as a single subretinal injection into the affected eye.
Side effects:
· Eye inflammation: The most common side effect of Luxturna is eye inflammation, which can cause redness, pain, light sensitivity, and blurred vision.
· Increased intraocular pressure: In some cases, Luxturna may raise intraocular pressure, which can lead to glaucoma.
· Retinal detachment: There is a risk of retinal detachment, a serious eye condition, following the injection.
Dose:
The recommended dose of Luxturna is a single injection into the affected eye.
Precautions:
· Consult a doctor: Before receiving Luxturna, consult with an ophthalmologist to ensure it is safe for you and to discuss potential risks and benefits.
· Eye exams: Regular eye exams are important to monitor the effectiveness of the treatment and detect any potential complications.
· Allergic reactions: Inform your doctor if you have any allergies to the components of Luxturna.