An eye-drop version of Krystal Biotech's skin gene therapy helped restore vision of boy with rare disease
A reformulated version of Krystal Biotech’s gene therapy for “butterfly skin” helped restore the vision of a boy with a rare disease, which can cause scarring of the eye tissue in some patients.
The 13-year-old boy — Antonio, according to the AP — has a rare genetic disease called dystrophic epidermolysis bullosa, or DEB, that disrupts the production of a type of collagen. It leads to skin that’s extremely fragile and that can blister or tear, like the wings of a butterfly. Around a quarter of these children also experience scarring of the eye, according to Krystal, which can potentially result in blindness.
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