Epidermolysis bullosa could soon get new kind of gene therapy

Wounds have been a constant fact of Aaron Owens’ life. His skin is so fragile that friction that would be trivial to others — rolling over in bed, weight shifting against the seat as the car turns — could scrape off his tissue. He didn’t like being in public sometimes because people would stare at the boy covered in bandages.

When he enrolled in a clinical trial a few years ago, the teenager didn’t know which wound on his body was treated with the experimental medication, and which was dosed with a placebo. This trial wasn’t like so many others where some enrollees get the drug in question and others get a sham dose. Rather, Owens received both, but on different wounds.