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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.

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