A devastating skin condition could soon get its first medication, as the frontiers of gene therapy expand

A devastating skin condition could soon get its first medication, as the frontiers of gene therapy expand

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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. Soon, though, Owens noticed that portions of skin in some areas started


to heal, and heal faster and stronger than he had experienced in his lifetime contending with these wounds. It was a clue that, maybe, the medication was working. STAT+ Exclusive Story


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