The FDA Just Approved Rub-On Gene Therapy That Helps “Butterfly” Children

May 23, 2023

(The Guardian) – 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 “butterfly children” like Anthony, the problem is that their bodies don’t make the type of collagen that holds the skin’s layers together. The result is chronic, blistering wounds—all over the skin, but also inside the throat and sometimes on the eyes. (Read More)

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