Thanks to gene therapy administered with eye drops, a young patient who was blind due to the consequences of a rare disease, dystrophic epidermolysis bullosa, recovered his sight in the treated eye. This is what was announced in the New England Journal of Medicine by Arianna Tovar Vetencourt of the Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine.
La therapy si chiama beremagene geperpavec (B-VEC; Vyjuvek).
Treatment with topical B-VEC applied directly to the right eye, visual acuity improved markedly for the 13-year-old boy, legally blind and with recessive dystrophic epidermolysis bullosa at age 7 years.
“Our data support further investigation of B-VEC in the care of patients with dystrophic epidermolysis bullosa with ocular surface involvement,” Vetencourt and colleagues wrote, acknowledging that larger studies and longer follow-up are needed. However, they continue, “the degree of improvement we observed was remarkable, given the lack of changes in the contralateral eye, which underwent similar treatment but without the ophthalmic application of B-VEC.”
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