Forbes May 24, 2023
Greg Licholai, MD

The Food and Drug Administration (FDA) has approved a transformational treatment for one of the most tragic of pediatric skin diseases. The first-of-its kind topical gene therapy for Epidermolysis bullosa (EB) is expected to be available in the United States in the third quarter of 2023 under the name is VYJUVEK according to Krystal Biotech (KRYS.O). EB is a rare, debilitating skin disorder characterized by fragile skin that blisters and tears easily. The young people affected by EB are sometimes called “Butterfly Children” because of their highly fragile skin. Current treatment options primarily focus on managing symptoms and preventing infection, but the groundbreaking gene therapy offers new hope.

“This approval marks what we believe is the beginning of the end...

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Topics: Biotechnology, FDA, Govt Agencies, Pharma / Biotech
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