First-of-its-kind gene therapy approved for healing wounds in “butterfly children”
Children born with a rare disease that makes their skin as fragile as a butterfly’s wings, leaving them vulnerable to frequent wounding and constant pain, will finally have access to a new treatment that aims to fix the genetic cause of the condition.
The therapy, known as Vyjuvek and made by Krystal Biotech, was approved by the FDA on Friday afternoon for treating patients six months or older with dystrophic epidermolysis bullosa, or DEB. The drug is a long-awaited solution for many families afflicted by the hellish condition, for which there are no approved treatments in the US.
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