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Retinal dystrophy causes blindness. Photo: MabelAmber
Retinal dystrophy causes blindness. Photo: MabelAmber

A Philadelphia company develops Luxturna, a therapy that cures a type of blindness

The new Luxturna therapy presented this week costs $850,000 and can end blindness for retinal dystrophy.

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Can you imagine the day when we will cure blindness? Well, imagine no more, because we are almost there at that moment. Although it is impossible to cure any type of blindness, the new Luxturna therapy could end the blindness caused by retinal dystrophy.

The company Spark Therapeutics has developed Luxturna. Located in Philadelphia, this company announced on January 3 that its Luxturna therapy had been approved to go on the market. With a price of $4,250,000 per eye, this therapy promises to restore vision to those affected by retinal dystrophy.

Retinal dystrophy

This type of condition is actually a hereditary disease. Retinal dystrophy affects between 1,000 and 2,000 people throughout the United States. The patients suffer the progressive death of the main cells of the eyes, the so-called photoreceptors. This leads to a progressive decrease in the visual capacity that can appear from the moment of birth or later in life, although it is more common during the first twenty years.

The results

Luxturna therapy has proven to be effective in the fight against blindness caused by retinal dystrophy. The published results show that more than nine out of ten patients benefited from the treatment.

The treatment consists of administering a new gene, the so-called RPE65, to act in place of the mutated gene that causes the visual blockage. The administration of this new healthy gene readies the body to deal with the problems of the visual cycle and gradually improve its functioning.

The price

A priori, the price may seem outrageously expensive. However, its announcement has been a surprise in the sector, which expects it to be even higher. This is due to the fact that it’s an innovative process that can help many people recover their sight. The techniques used for its development are very expensive and there has been extensive research behind. In addition, the spectrum of patients, compared with other diseases, is not very high.

Aware that many patients will not be able to cover this expense, Spark Therapeutics is negotiating with the United States health service the option to pay the treatment in installments. We will have to wait to see what the financing plans are.

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