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A boy recovers his sight with gene therapy eye drops

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A boy recovers his sight with gene therapy eye drops

Young patient blind from rare disease recovers sight with gene therapy

A breakthrough in gene therapy has led to a young patient recovering his sight after being blind due to the rare disease dystrophic epidermolysis bullosa. The treatment, administered with eye drops, has shown remarkable results in improving visual acuity for the 13-year-old boy.

The therapy, called beremagene geperpavec (B-VEC; Vyjuvek), was applied directly to the patient’s right eye, leading to significant improvement in his vision. The patient had been legally blind and diagnosed with recessive dystrophic epidermolysis bullosa at the age of 7.

Arianna Tovar Vetencourt of the Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine announced the results in the New England Journal of Medicine. The study, conducted by Vetencourt and colleagues, has shown promising results in the treatment of patients with dystrophic epidermolysis bullosa with ocular surface involvement.

The researchers noted the need for larger studies and longer follow-up to further investigate the potential of B-VEC in treating this rare disease. However, they highlighted the remarkable degree of improvement observed in the patient who underwent the treatment, compared to the contralateral eye which did not receive the ophthalmic application of B-VEC.

The news of this groundbreaking therapy offers hope for patients suffering from rare diseases and paves the way for further advancements in gene therapy. The full article can be found on ANSA.it.

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