Aggregated News
Antonio Vento is 13 years old. He’s a tiny figure in bandages who doesn’t walk and, until recently, couldn’t see more than shadows. He has dystrophic epidermolysis bullosa, an inherited disease that makes his skin so fragile that kids with the illness are called “butterfly children.”
But now, thanks to a novel gene therapy squirted onto his skin and dripped into his eyes, things are better. His wounds have gotten smaller, and a visit to the eye doctor this week confirmed that his vision had dramatically improved.
“They said my right eye is 20/25,” he chirped in Spanish during a phone call. “Now I can see small things.” That includes the blocks and items in the video game Minecraft, which he has started to play.
And call him Anthony, he said. He prefers it.
On Friday, the US Food and Drug Administration approved the gene-replacement treatment Anthony received, making it the first gene therapy for sale that is applied to the outside of a patient’s body—as well as the first intended to be used on the same person repeatedly.
For...